Development and Sensibility Evaluation of the Gait Outcomes Assessment List (GOAL) Module for Children with Lower Limb

Anomalies

by

Jennifer Anne Dermott

A thesis submitted in conformity with the requirements for the degree of Master of Science

Rehabilitation Sciences Institute University of Toronto

© Copyright by Jennifer Anne Dermott, 2015

ii

Development and Sensibility Evaluation of the Gait Outcomes Assessment List (GOAL) Module for Children with Lower Limb

Anomalies

Jennifer Anne Dermott

Master of Science

Rehabilitation Sciences Institute

University of Toronto

2015

Abstract

Background: Functional evaluation of children with lower limb anomalies (LLAs) is limited

due to a lack of appropriate outcome measures for this population.

Objective: To develop a module of the Gait Outcomes Assessment List (GOAL) questionnaire

that is specific and sensible for use with children with LLAs.

Methods: An iterative adaptation and sensibility evaluation of the GOAL was informed by

field-testing this questionnaire with children and their parents, conducting cognitive interviews

with children, and administering an e-Survey to healthcare professionals.

Results: Twenty-five children, 20 parents, and 31 healthcare professionals evaluated the content

and sensibility of the GOAL that ultimately led to 22 of the original 50 items being retained, 16

modified, and six new items added in the final questionnaire iteration (GOAL-LLA 2.0).

Conclusion: This study supports the GOAL-LLA 2.0 as a promising new outcome measure for

comprehensively evaluating the function and priorities of children with LLAs, for both research

and clinical purposes.

iii

Acknowledgments

I would like to thank Unni Narayanan, who, when I first approached him with the idea of

pursuing a graduate degree, responded with enthusiasm, support, and a number of project ideas.

Unni was instrumental in setting me on my path. His commitment to providing children and

families with an opportunity to have a voice is inspiring.

I am grateful to Virginia Wright for agreeing to co-supervise my project. Virginia’s contribution

to my development as a researcher has been exceptional. Regardless of her own grant deadlines

and work demands, or where she was in the world, she could be relied upon for a quick reply.

She was generous with her time and her words of encouragement. I will endeavor to emulate her

attention to written detail.

Nancy Salbach was the ideal addition to my advisory committee, introducing an academic rigour

to my predominately clinical way of thinking. Nancy’s suggestions always improved the depth

and quality of my work. I greatly appreciate that, despite being on sabbatical, Nancy was always

invested in my work, providing timely and thoughtful feedback.

I am eternally grateful to my husband Dean, for without his unwavering support I would never

have been able to complete a thesis, work full-time, and raise a family. To my extended family,

who has always stood by with love and support, and provided an extra hand when needed. To my

sons, Jack and Sam, I hope I have been able to exemplify the merits of working hard to obtain

your goals. The past couple years has not been easy for any of us but the fact is, we survived and

I feel we are actually the better for it.

I would like to acknowledge support through Holland Bloorview Graduate Support program,

SickKids Continuing Professional Development Fund, and the University of Toronto Open

Fellowship. I would also like to acknowledge Unni’s research team (Shannon Weir, Clarissa

Encisa, and Rachel Moline) who always responded to my questions so promptly, and his past

students (Benjamin Davidson, Falisha Karpati, and Edwin Ho) for their foundational GOAL-CP

work.

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Table of Contents

Table of Contents ........................................................................................................................... iv

List of Tables ................................................................................................................................ vii

List of Figures .............................................................................................................................. viii

List of Appendices ......................................................................................................................... ix

List of Abbreviations ...................................................................................................................... x

Chapter 1 Introduction .................................................................................................................... 1

Chapter 2 Lower Limb Anomalies ................................................................................................. 3

2.1 Characterization and Epidemiology ..................................................................................... 3

2.2 Treatment ............................................................................................................................ 3

2.2.1 Rationale and Goals of Treatment .......................................................................... 4

2.2.2 Conservative (Non-surgical) Management Options ............................................... 5

2.2.3 Surgical Options ...................................................................................................... 5

Chapter 3 Outcome Measurement in Children with Lower Limb Anomalies ................................ 8

3.1 The International Classification of Functioning, Disability, and Health ............................ 8

3.1.1 Linking Health-related Measures to the International Classification of

Functioning, Disability, and Health ........................................................................ 9

3.2 Current Outcome Measurement in Children with Lower Limb Anomalies ..................... 10

3.2.1 Technical Outcomes .............................................................................................. 10

3.2.2 Functional Outcomes ............................................................................................ 11

Chapter 4 Gait Outcomes Assessment List (GOAL) .................................................................... 19

4.1 Background and Rationale ................................................................................................ 19

4.2 Application of the Gait Outcomes Assessment List to Children with Lower Limb

Anomalies- Sensibility ...................................................................................................... 22

Chapter 5 Rationale and Study Objectives ................................................................................... 25

5.1 Rationale ........................................................................................................................... 25

v

5.2 Purpose .............................................................................................................................. 26

5.2.1 Objectives ............................................................................................................. 26

Chapter 6 Methods ........................................................................................................................ 28

6.1 Study Design ..................................................................................................................... 28

6.2 Child and Parent Perspectives (Phase 1) ............................................................................ 29

6.1.1 Participants ............................................................................................................ 29

6.1.2 Recruitment ........................................................................................................... 29

6.1.3 Data Collection ..................................................................................................... 30

6.1.3.1 Questionnaire Administration (Objectives 1, 3, and 4) ........................................ 30

6.1.3.2 Cognitive Interviews (Objectives 1 and 2) ........................................................... 31

6.1.4 Analysis of Child and Parent Perspectives (Phase 1) ........................................... 32

6.1.5 Child and Parent Sample Size ............................................................................... 34

6.2 Health Care Professional Perspectives (Phase 2) .............................................................. 34

6.2.1 Participants ............................................................................................................ 34

6.2.2 Sampling Recruitment .......................................................................................... 34

6.2.3 Data Collection ..................................................................................................... 35

6.2.3.1 e-Survey Questionnaire (Objective 1 and 2) ......................................................... 35

6.2.4 Analysis of Healthcare Professional Perspectives ................................................ 35

6.2.5 Healthcare Professional Sample Size .................................................................... 36

Chapter 7 Results .......................................................................................................................... 37

7.1 Child and Parent Perspectives (Phase One) ...................................................................... 37

7.1.1 Participants ............................................................................................................ 37

7.1.2 Content Adaptation (Objective 1) ......................................................................... 39

7.1.3 Sensibility Evaluation (Objective 2) ..................................................................... 47

7.1.4 Children versus Parent Response (Objective 3) .................................................... 49

7.1.5 Item Scores versus Importance Ratings (Objective 4) .......................................... 50

vi

7.2 Healthcare Professional Perspectives (Phase Two) .......................................................... 51

7.2.1 Participants ............................................................................................................ 51

7.2.2 Content Adaptation (Objective 1) ......................................................................... 51

7.2.3 Sensibility (Objective 2) ....................................................................................... 55

7.3 GOAL-LLA 2.0 ................................................................................................................ 56

Chapter 8 Discussion .................................................................................................................... 57

8.1 Limitations ......................................................................................................................... 63

8.2 Future Directions .............................................................................................................. 66

8.3 Conclusions ....................................................................................................................... 67

References ..................................................................................................................................... 68

Appendices .................................................................................................................................... 77

vii

List of Tables

Table 1. Patient Reported Outcome (PRO) Measures Identified ................................................. 14

Table 2. Categorization of the Items in the GOAL and the Outcome Measures Identified in

Published Paediatric LLA-related Studies, using the ICF Framework ......................................... 22

Table 3. Phase 1 Participants by Questionnaire Version Administered ....................................... 37

Table 4. Child Participant Demographics .................................................................................... 38

Table 5. Summary of Content Adaptation from the Initial to Third Iteration of the GOAL ....... 39

Table 6. Results of the Initial Administration of the GOAL (GOAL-CP) in Phase 1 ................ 41

Table 7. Results of the Second Administration of the GOAL (GOAL-LLA 1.0) in Phase 1 ...... 44

Table 8. Demographics of Phase 2 Respondents ......................................................................... 51

Table 9. Summary of Content Adaptation from the Third to Final Iteration of the GOAL ........ 51

Table 10. Results of the Administration of the GOAL-LLA 1.1 in Phase Two .......................... 53

viii

List of Figures

Figure 1. International Classification of Functioning, Disability, and Health .............................. 9

Figure 2. Priority Framework for Outcomes Assessment ............................................................ 20

Figure 3. Example Scale Used in GOAL Domains Evaluating Self-perceived Ability............... 21

Figure 4. Example Scale Used in GOAL Domains Evaluating Emotions ................................... 21

Figure 5. Illustration of Study Events ......................................................................................... 28

Figure 6. Modified Scale used in GOAL Domains Pertaining to Self-perceived Ability ........... 49

Figure 7. Children's and Parents' Mean Item Scores for the GOAL-LLA 1.1 ............................. 49

Figure 8. Children’s and Parents’ Mean Importance Ratings for the GOAL-LLA 1.1 ............... 50

ix

List of Appendices

Appendix 1. Literature Search Strategy ....................................................................................... 77

Appendix 2. Development of the Gait Outcomes Assessment List (GOAL) ............................. 78

Appendix 3. GOAL-CP ............................................................................................................... 80

Appendix 4. Child Consent Form ................................................................................................ 89

Appendix 5. Determination of Child’s Capacity to Consent ....................................................... 93

Appendix 6. Research Ethics Board Approval ........................................................................... 94

Appendix 7. Research Ethics Board Approval- Holland Bloorview ........................................... 95

Appendix 8. University of Toronto Study Approval ................................................................... 96

Appendix 9. Information Sheet .................................................................................................... 97

Appendix 10. Cognitive Interview Guide .................................................................................... 98

Appendix 11. Cognitive Pretesting Model ................................................................................. 104

Appendix 12. Information Letter for Healthcare Professionals ................................................. 105

Appendix 13. GOAL-LLA 1.0 ................................................................................................... 107

Appendix 14. GOAL-LLA 1.1 ................................................................................................... 117

Appendix 15. Results of the Third Administration of the GOAL (GOAL-LLA 1.1) in Phase 1

..................................................................................................................................................... 127

Appendix 16. GOAL-LLA 2.0 ................................................................................................... 129

x

List of Abbreviations

AAOS: American Academy of Orthopedic Surgeons

ADL: Activities of Daily Living

AMP: Amputee Mobility Predictor

AOFAS: American Orthopedic Foot and Ankle Society

ASK: Activities Scale for Kids

BSCOS: British Society for Children’s Orthopaedic Surgery

CAPP-FSI: Child Amputee Prosthetics Project- Functional Status Inventory

CHQ: Child Health Questionnaire

CORA: Center of Rotation of Angulation

COSMIN: Consensus-based Standards for the selection of health status Measurement

Instruments

CP-QOL: Cerebral Palsy Quality of Life

CP: Cerebral Palsy

CPT: Congenital Pseudarthrosis of the Tibia

CT: Computed Tomography

EBM: Evidence-based Medicine

EFT: External Fixator Treatment

EPIC: Evidence-Based Practice Confidence Scale

FAQ: Gillette Functional Assessment Questionnaire

FMS: Functional Mobility Scale

GDI: Gait Deviation Index

GGI: Gillette Gait Index

GMFCS: Gross Motor Function Classification System

GMFM: Gross Motor Function Measures

GOAL-CP: Gait Outcomes Assessment List for Cerebral Palsy

GOAL-LLA: Gait Outcomes Assessment List for Lower Limb Anomalies

GPS: Gait Profile Score

GRF: Ground Reaction Force

HCP: Health Care Professional

HDQ: HIV Disability Questionnaire

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ICC: Intraclass Correlation Coefficient

ICF: International Classification of Function, Disability, and Health

LLA: Lower Limb Anomaly

LLD: Leg Length Discrepancy

LLRS: Limb Lengthening and Reconstruction Society

MAD: Mechanical Axis Deviation

MAP: Motion Analysis Profile

MDC: Minimal Detectable Change

mLDFA: Mechanical Lateral Distal Femoral Angle

MPTA: Medial Proximal Tibial Angle

MSTS: Musculoskeletal Tumour Society Rating Scale

PedsQL: Pediatric Quality of Life

PODCI: Pediatric Outcomes Data Collection Instrument

PRO: Patient-Reported Outcome

QAPAQ: Qualitative Assessment of Physical Activity Questionnaire

QOL: Quality of Life

ROM: Range of Motion

SAC: Scientific Advisory Committee

SD: Standard Deviation

SEM: Standard Error of Measurement

TESS: Toronto Extremity Salvage Scale

TSF: Taylor Spatial Frame

1

Chapter 1 Introduction

The purpose of this thesis is to address the need for a comprehensive outcome measure to

evaluate the function (physical and psychosocial) of children with lower limb anomalies (LLAs)

(i.e., orthopaedic impairments that are characterized by deformity, deficiency, and/or length

discrepancies).

The altered structure of the lower limb often presents physical challenges related to a

pathologically abnormal gait, but may also have a social and psychological impact. Outcomes

research related to children with LLAs tends to focus on correction of the impairment, namely

technical outcomes (at the level of body functions and structure) including limb alignment and

length discrepancy, measured radiographically, and post-operative complications and healing

index (1). Although functional (physical and/or psychosocial) improvement is often considered

an aim and important outcome of surgery, a comprehensive measurement of function (at the level

of activity and participation) is rarely reported (2). Indeed, when currently available functional

outcome measures have been used in this population they have proved problematic,

demonstrating limited discriminative ability and an inability to detect change (2-6). For this

reason, some investigators have opted to use non-validated functional status measures they have

created to fulfill their study’s measurement requirement (5, 7-9).

This imperative to develop a standardized functional outcome measure for use with this

population has been identified by a number of paediatric LLA research groups so as to improve

the uniformity and comprehensiveness of patient assessment, to ensure comparable

benchmarking across intervention studies, and ultimately to provide evidence that will give

insight into the assumed functional benefits of treatment (10-13). Moreover, an outcome measure

that identifies patient priorities would facilitate treatment planning that addresses the specific

goals and expectations of each family, as well as align with the philosophy of evidence-based

medicine (EBM), defined as “the integration of best research evidence with clinical expertise and

patient values” (14). If clinical decisions are to serve the patient, their unique preferences,

concerns, and expectations must be integrated (14). To date there are no studies that use

individualized measures to explore children’s or families priorities with regard to treatment of

LLAs.

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The following sections provide background information about the conditions that result in LLAs,

the functional impact of these, and the treatment options that are available. A framework to

conceptualize function (ICF) will be described. Common outcome measures (technical and

functional) that have been used for children with LLAs, and how these measures fit within this

framework, will be presented. The literature will be reviewed to highlight the gap in functional

outcome measurement, based on the infrequent and problematic use of patient-reported outcome

(PRO) measures for this population. Finally the Gait Outcomes Assessment List (GOAL) will be

introduced and the plans to evaluate and adapt this questionnaire to create a module specific for

children with LLAs will be detailed.

3

Chapter 2 Lower Limb Anomalies

2.1 Characterization and Epidemiology

Lower limb anomaly (LLA) is a broad term that encompasses length discrepancy with or without

limb deficiency (missing or insufficiently developed parts), and/ or deformity (malalignment) of

the lower limb/s. LLAs often present as a structural impairment associated with deficits in

strength and range of motion, and consequently, altered gait mechanics. In terms of specific

types of LLA, lower limb length discrepancies are common, with 23% of the general population

having a discrepancy of 1 centimetre or greater (15), although as the magnitude of difference

increases, the prevalence decreases (3). Large magnitude discrepancies (greater than 1.5

centimetres) occur in about 5% of the population (1). Lower limb deficiencies have a reported

incidence of 2 to 7 in 10 000 births (16). There is a wide spectrum of lower limb deficiencies that

include small or deformed feet, absent or deficient bony segments in the feet, lower leg or thigh,

unstable joints due to absent or deficient ligaments, absent or weak muscles, and inequalities of

length and girth (1, 17). Congenital deficiencies account for approximately two thirds of lower

limb amputations (12). Fibular deficiency is the most common long bone deficiency with an

incidence of 7-20 per million births (18). LLAs might also be acquired as a result of bone loss,

malunion, or abnormal growth following trauma or post-traumatic sequelae, oncologic disease,

or infection (10, 19). A lower limb deformity is characterized by abnormal alignment of the

thigh (femur) and/or lower leg (tibia and fibula) and/or foot and ankle. These deformities might

occur in one or more of the coronal, sagittal, and/or axial (transverse) planes. It is important to

note that these different types of LLAs often coexist. For example, a length discrepancy might

be secondary to a limb deficiency; and may be associated with a deformity that could be in more

than one plane. More details about specific LLAs are provided below in the context of treatment

options (Section 2.2).

2.2 Treatment

The prevailing management philosophy is to correct the LLA, and to this end, children with

LLAs may undergo a multitude of orthopaedic surgeries throughout their childhood. The typical

model of care for the management of these children positions the orthopaedic surgeon as the key

4

healthcare professional (HCP) to inform treatment, with input from other members of the

healthcare team (i.e., nurse, physical therapist, orthotist/prosthetist). If the healthcare team feels

there is a need, the circle of care might be expanded to include a social worker, or a psychologist.

This is because there may be an additional effect of an LLA on an individual’s social and

psychological function. HCPs and families must collectively decide the most appropriate

treatment option (or combination of options) for the individual child. Families rely on HCPs to

provide information related to the rationale and the details of available treatment options

(detailed below).

2.2.1 Rationale and Goals of Treatment

LLAs often result in an abnormal gait pattern and increased mechanical effort (2, 20-22).

Treatment of LLAs aims to minimize these consequences through surgical and non-surgical

techniques (as described in 2.2.2) to level the pelvis, improve symmetry, and allow more even

distribution of joint loads in a bipedal standing position and during stance phase in gait (1). If

uncorrected, compensatory strategies are likely adopted by the child in an attempt to normalize

gait patterns, provide a more stable base of support, optimize energy expenditure, and reduce

joint load (22). The long-term sequelae of these compensatory strategies may include pain, joint

degeneration, and dynamic deformity of adjacent joints (22). Incremental increases in

discrepancy increase the stress across the sacroiliac joint (23). There is general consensus that

in the absence of a deformity, leg length discrepancies less than 2 cm do not require treatment as

these have negligible clinical impact (24, 25). Deformity can cause asymmetry of loading across

all lower extremity joints. Asymmetric loading across the knee joint is a well-recognized risk

factor for the development of osteoarthritis (26, 27).

These gait-related consequences of an LLA not only affect a child physically but may also have a

social and psychological impact. For example, fatigue related to increased mechanical effort,

functional impairments (e.g., limited range of motion), or dissatisfaction with appearance can

limit participation in social and physical activities and can lead to difficulties in social and

psychological adjustment (2, 3, 20).

5

2.2.2 Conservative (Non-surgical) Management Options

Modest leg length discrepancies of less than 6% inequality are amenable to non-operative

treatment (28). Shoe inserts (insoles) can be used to equalize discrepancies up to 1.5

centimetres, and if further correction is needed, the sole of the shoe can be built up externally

(29). It is recommended that external shoe lifts not exceed five centimetres, to minimize the risk

of an ankle sprain, although, if required, an ankle-foot orthosis can provide support to an

unstable joint (30). It is estimated that about 1 in 1000 individuals in the general population have

been prescribed a shoe lift (1). For discrepancies greater than two centimetres, these approaches

become less acceptable as a long-term solution because of the perceived aesthetics including

dissatisfaction with shoe choice, impaired biomechanics and barefoot function, and associated

costs of maintenance and replacement of devices and footwear (1). As a result, individuals are

more likely to either live with their asymmetry or opt for surgical intervention. For massive

length discrepancies, typically related to severe limb deficiencies for which surgical management

might not be feasible, prosthetics (e.g., extension prosthesis) can be used. There are no effective

non-surgical strategies to deal with clinically significant deformities (malalignment) of the lower

limbs. Deformities of the foot can be accommodated with custom orthotics or orthopaedic

footwear.

2.2.3 Surgical Options

2.2.3.1 Limb Reconstruction

There are a number of surgical options to manage limb length discrepancies, limb deformities,

and some limb deficiencies. Limb length discrepancies can be managed by complete

epiphysiodesis (growth plate arrest) or shortening procedures of the longer side, or lengthening

procedures by gradual distraction osteogenesis of the bone/s on the short side using lengthening

devices such as external fixators (EFT) or motorized intramedullary rods. Lower limb deformity

can be corrected using guided growth techniques by temporary or permanent hemiepiphysiodesis

(partial growth arrest), acute deformity correction by osteotomies, or gradual correction by

distraction osteogenesis using external fixators. Many LLAs are managed by various

combinations of the above.

Epiphysiodesis is arrest of the growth plate on the longer side, allowing for the shorter side to

catch up, and is considered an option for projected length discrepancies between two and five

6

centimetres at skeletal maturity (31) or in combination with lengthening techniques to reduce the

magnitude or number of lengthening procedures of the shorter side. Epiphysiodesis does limit a

child’s final standing height therefore it is less likely to be used in conditions associated with

short stature (e.g., Noonan’s syndrome, Turner’s syndrome) or in isolation for more significant

length discrepancies. This is considered a technically simple procedure to perform but must be

appropriately timed, to correlate with remaining growth if it is to be successful. Epiphysiodesis

is no longer an option if there is too little growth remaining (near skeletal maturity) or if the short

limb cannot grow due to growth arrest.

External fixator treatment is used for lengthening and gradual deformity correction using

distraction osteogenesis. Bone is lengthened and or alignment corrected through an osteotomy

that is gradually pulled apart using an external fixator that uses pins and wires to anchor the bone

segments above and below the osteotomy. The rate of distraction of the bone and soft tissues

(including muscles and neurovascular structures) is gradual enough to be tolerated by the patient

and to allow for new bone formation in the gap (regenerate) that is being created at the site of the

osteotomy. There are two categories of external fixators: a monolateral fixator that allows for

lengthening but is less versatile for deformity correction, and a circular or ring-type fixation that

allows for multiplanar correction in addition to lengthening (32).

The maximum amount of length that should be targeted in a single lengthening procedure has

traditionally been no more than 20% of the limb segment length (33). Lengthening beyond this

increases the likelihood of complications including functional deficits related to muscular and/or

neural tissue dysfunction and deformity at adjacent joints (34).

Lengthening may also be performed over an intramedullary nail in conjunction with an external

fixator. The benefit of this technique is that it decreases time in the external fixator by providing

increased stability to the consolidating bone, decreasing the risk of fracture post-frame removal

(9, 35). The possible drawback of this technique is increased risk of deep infection (35). The

possible functional benefit has not been investigated.

Recently lengthening through internal, intramedullary devices has been trialed. Lengthening is

actualized through a mechanical, motorized, or electro-magnetic system. This fully implantable

method of lengthening is becoming more widely available but has not been adequately studied to

7

compare the safety, efficacy, and cost with conventional methods (36). The functional impact of

internal versus external lengthening has not been explored.

2.2.3.2 Amputation

Amputation was once considered standard procedure for severe congenital limb deficiencies in

which the length discrepancy was more than 5 centimetres at birth or predicted to be greater than

15% at skeletal maturity, if there were less than three rays in the foot, and if the foot was unable

to achieve a plantigrade position (8, 28). The decision to amputate is commonly made in infancy

and performed at about the time a child begins to stand (28). Although surgical advances now

permit limb preservation to be considered in cases where amputation would have once been

routine, a non-functional foot continues to be an indication for a Boyd or Syme amputation (37).

Effort is made to achieve an optimal residual limb for prosthetic fitting, which in children may

necessitate additional surgical intervention to account for growth. In a study by Westberry et al.

(38), 117 Boyd procedures were performed on 109 children with approximately one quarter of

these requiring concomitant procedures such as a proximal tibial epiphysiodesis, and one third

requiring subsequent surgical procedures.

Van Nes rotationplasty is an alternative amputation technique that may be considered for femoral

deficiencies. This involves rotation of the lower limb, allowing the ankle to foot to act as a

functional knee joint within a prosthesis. This procedure offers a functional “knee” joint, less

energy consumption and potentially fewer future surgeries but is not always well accepted by

families, because of the cosmetically unusual appearance (39).

If limb preservation (i.e., reconstruction) can be done safely and reliably, many orthopaedic

surgeons consider amputation a last resort (40), even though, the long-term functional outcome

of limb preservation, relative to the previously routine amputation, is not as well documented

(41).

8

Chapter 3 Outcome Measurement in Children with Lower Limb Anomalies

Once the details of the treatment options are well understood by families, they must decide what

is “best” for their child. Families will look to the HCP for guidance related to the expected

outcomes and effectiveness of treatment in achieving these outcomes. As such, there is an

imperative to focus on the measurement of outcomes. As highlighted previously, LLAs are

likely to impact children’s lives on multiple levels (i.e., physical and psychosocial functioning)

so a measurement tool that is designed to capture these levels is necessary to evaluate the

outcomes of treatment.

3.1 The International Classification of Functioning, Disability, and Health

The ICF conceptualizes function as a “dynamic interaction between a person’s health condition,

environmental factors, and personal factors” (42). This framework provides a standard language

with which to communicate and describe the effects of a health condition on one’s functional

ability (43). It defines function as more than traditionally measured structural impairments, and

focuses on how individuals live with their condition (44).

The individual components of the ICF include: body structures (anatomical parts of the body),

body functions (body systems including psychological function), activities (the completion of a

specific task or action), and participation (activities that are performed for a specific purpose or

role), environment (attitudinal, physical, and social), and personal factors (45). Environmental

and personal factors are collectively referred to as contextual factors and can be either facilitators

or barriers to overall function. The ICF illustrates a dynamic and complex multidirectional

interaction between all of its components (Figure 1), suggesting a linear relationship may not

exist between a discrete area of the ICF and overall functional status (44) or between changes

among ICF areas (46). This suggests that outcomes such as range of motion (body function) or

limb alignment (body structure), that are traditional clinician measures of impairment associated

with LLA, may not be valid proxies for functional status. Moreover, one cannot assume that

changes in these outcomes as a result of intervention, will result in the desired changes in other

areas of the ICF.

9

Figure 1. International Classification of Functioning, Disability, and Health (WHO, 2001)

For patients, the most meaningful indicators of the effectiveness of interventions must be tied to

the desired goals of the patient (47). These are more likely to focus on improvements in

activities and participation affected by the condition rather than indicators that focus on

improvements in body structure and function (47), termed technical outcomes by Goldberg (48).

Narayanan suggests that the ultimate desired functional outcomes (focused on activities and

participation) might not require technical perfection (47). For example, maintaining a length

discrepancy up to one centimetre post-lengthening may be advantageous for individuals with an

equinus contracture of the ankle joint to minimize the risk of tripping.

3.1.1 Linking Health-related Measures to the International Classification of Functioning, Disability, and Health

Specific linking rules have been developed to connect health-related measures to the ICF (49,

50). Every item within a measure can be linked to the content coding that has been built into the

ICF classification, and thus also connected to a specific domain of the ICF model. Any item that

does not fit into a domain is classified as ‘not definable’. Personal factors are not coded in the

ICF classification, therefore, were not included in the original linkage rules published in 2002

(50). Three years after being introduced, the linkage rules were revised to increase specificity,

allowing differentiation from other ‘not covered’ concepts (49). This adjustment improves

consistency with the ICF model (49). Once completed, this linkage process illustrates how well

an outcome measure relates to the ICF and also highlights what concepts within the ICF a

specific measure is focused on. This allows for easier comparison among measures and should

10

facilitate researchers and clinicians in selecting the most appropriate measure to meet their needs.

The ICF linkage process is illustrated in section 4.2.

3.2 Current Outcome Measurement in Children with Lower Limb Anomalies

LLA-related literature consists primarily of case series and observational comparative studies of

surgical interventions, with a multitude of outcomes measured. Orthopaedic outcome

measurement has been categorized as either technical (at the level of body functions and

structures) or functional (at the level of activities and participation, environmental and personal

factors) (47).

3.2.1 Technical Outcomes

Traditionally, studies on LLAs provide thorough reports of radiographic outcomes (10). Indeed,

surgical success is often determined radiographically, with the objective being a +/- 5 millimetre

residual length difference and a mechanical axis deviation (MAD) through the centre of the knee

or comparable to the contralateral limb (1, 9, 51-56). Additional components of a deformity

analysis include: centre of rotation angulation (CORA), and standard measures of joint

orientation: mechanical lateral distal femoral angle (mLDFA), medial proximal tibial angle

(MPTA) (1). Femoral and tibial torsion may be assessed on computed tomography (CT) (57),

although more recently, EOS has been validated as a good alternative with equivalent reliability

and accuracy, and a comparatively reduced dose of radiation (58). As suggested previously,

success ascertained radiographically cannot be assumed to result in a successful outcome as

reflected across the other levels of the ICF encompassing physical and psychosocial function.

These “outcomes” reported are related to the efficiency or time needed for treatment rather than

true measures of treatment effectiveness. These are typically based on radiographic evaluation,

of which the most widely used is known as the healing index (1). In the context of limb

lengthening, the healing index is reported as the distraction-consolidation time (in days or

months) required per centimetre of length achieved. Other related numeric measurement

parameters include lengthening time, distraction index, mean time to consolidation, and

weightbearing index (59).

11

Complication rates, which are common, are routinely reported, often using Paley’s classification

of difficulties during limb reconstruction as problems, obstacles, or complications (60).

Clinical impairment measures include joint range of motion (ROM) or strength assessment (1, 9,

51, 52, 54, 61). Measuring ROM during EFT is important for a variety of reasons, as

maintaining adequate ROM throughout EFT prevents muscular atrophy, diminishes joint reaction

forces, avoids cartilage damage, decreases pain, prevents adhesions of muscles to bony

regenerate, and increases blood supply to the bone, inducing bone formation (1). However, as

described earlier, ROM is not a sufficient proxy for overall functional status.

3.2.2 Functional Outcomes

Activity-based clinical outcomes include timed walk tests [e.g., 6- minute walk test (62), Timed

Up and Go (63)] or gait assessment. Assessing gait might involve an observational evaluation

by the surgeon or physical therapist (PT), sometimes as basic as characterizing a limp on clinical

evaluation (9) or noting independent versus assisted gait (61). Conversely, this could involve an

instrumented approach in a motion laboratory to provide a more detailed analysis of gait and

running mechanics, standing balance and postural sway (29). There is little consistency across

studies in the methodology employed in gait analysis, making interpretation and comparison of

results difficult (29). Furthermore, regardless of the approach taken, these measures provide no

direct information on the impact that improving gait speed or symmetry has on the overall

functional status of the individual.

3.2.2.1 Patient Reported Outcome (PRO) Measures

PRO measures allow for direct subjective assessment by the patient on elements of their health

and related function (64) and are considered the best way to measure participation domains of the

ICF (47). It is the individual who has first-hand knowledge of their daily functioning in all

contexts (65), making them the most qualified to report. Narayanan (47) states: “to the extent

that the patient’s perspective is recognized to be pre-eminent in making judgments about

effectiveness, the use of patient-reported outcome (PRO) measures is now considered the

standard when evaluating effectiveness of interventions”.

Despite the widespread use of PRO measures in many areas of health research and clinical care,

the use of PRO measures in pediatric orthopaedics lags behind (66) and was essentially non-

12

existent in all orthopaedic research prior to the 1980’s (67). It is argued that PRO measures are

more difficult to measure and interpret than traditional clinician measured “objective” parameters

(67). However, a major disadvantage of relying solely on traditional clinical outcomes in

evaluative orthopaedic research is that these may not directly relate to patients’ reasons or goals

for undergoing surgery (68, 69). The measured variables may be of interest to the clinician but

not necessarily to the patient being evaluated in the study (67). Furthermore, traditional clinical

outcomes have not always been found to correlate well with PROs (70, 71).

PRO measures can be either generic or condition-specific. In general, condition-specific

measures, designed to focus on issues relevant to a particular condition, are more sensitive to

change than generic ones (72), although the applicability of these measures is limited to the

diagnostic group for which they were designed. There are condition-specific measures available

for use with children that use a prosthetic device [e.g., Child Amputee Prosthetics Project-

Functional Status Inventory (CAPP-FSI), Amputee Mobility Predictor Questionnaire (AMP)].

Similarly, there are condition-specific measures available for use with children that have an LLA

related to sarcoma [e.g., Toronto Extremity Salvage Scale (TESS), Musculoskeletal Tumour

Society Rating Scale (MSTS)]. Most of these condition-specific instruments were developed for

an adult population and there is not always an equivalent measure for pediatrics. A pediatric

version of the TESS (pTESS) has been developed and a multi-centre study is presently

evaluating its psychometric properties (J. Piscione and S. Hopyan, personal communication,

September 14, 2014).

Individualized measures are a distinct type of PRO measures. To date there are no studies that

use these measures to explore children’s or families’ priorities in regard to treatment of LLAs.

There is increasing recognition that children should be consulted on their treatment goals and this

information incorporated into treatment plans (45, 73). Individualized measures allow for these

goals to be captured and measured (74). The initial administration of an individualized measure

requires the child or parent (or clinician) to identify intervention goals with subsequent

administration measuring change that is unique to the individual. Patients find outcomes are

most meaningful when they are aligned with their priorities (47) and attention to patient needs

and circumstances when planning care is associated with improved health care outcomes (75).

13

Examples of individualized measures include the Canadian Occupational Performance Measure

(COPM), the most frequently used individualized measure in paediatric rehabilitation (76), and

the Goal Attainment Scale (GAS). Both these measures were designed for an adult population

but have been used in paediatric studies with success (77). Tam et al (77) found that when these

individualized measures were used in pediatric research, most authors (19 out of 28 studies in

which COPM was used, 22 out of 34 GAS use studies) also employed a standardized fixed item

functional measure to evaluate the effectiveness of intervention.

In general, there are very few studies pertaining to children with LLAs that actually employ a

PRO measure. A literature search was conducted prior to commencement of this project with the

sole purpose of identifying validated PRO measures that have been used in these children.

Medline (1995 to 2013) was used as the source database. The index terms “leg length

inequality”, “limb deformity”, “osteogenesis distraction”, “bone lengthening”, “external fixator”,

“Ilizarov technique”, “femur”, “tibia”, “genu varum” or “genu valgum”, “amputation”, and

“artificial limb” were used in combination with “questionnaire” or the keyword “function”.

Inclusion criteria for detailed review of a publication included: the mean age of participants was

less than 18 years and no participants had upper extremity involvement. See Appendix 1 for the

full search strategy.

The search returned 435 papers, of which six were relevant. It is important to stress that

amputee-, sarcoma-, or joint-specific outcome measures (e.g., CAPP-FSI, MSTS or American

Orthopedic Society Foot and Ankle Society Score (AOFAS)) that were revealed in the search

were not included as the aim was to identify measures that would be applicable across the

spectrum of conditions that result in LLAs. Table 1 lists the six PRO measures identified within

these papers. There were no individualized measures used in any of the identified studies.

14

Table 1. Patient Reported Outcome (PRO) Measures Identified

in LLA-related Literature (1995-2013)

Name Construct Target

Population

Studies Cited

Child Health

Questionnaire (CHQ)

Physical and

psychosocial well-

being of children

(Generic)

Child report for ages

11-17 years; parent

report for ages 2

months up to 5 years

and 5-17 years

The effect of leg

length difference on

health related quality

of life: is the '2cm

rule' appropriate? (3)

Gillette Functional

Assessment

Questionnaire (FAQ)

Functional walking

ability and mobility

skills

(Originally

developed for CP)

Parent report for ages

2+ years

Measurement of

health-related quality

of life in children

undergoing external

fixator treatment for

lower limb

deformities (2)

Activity Scale for Kids

(ASK)

Measure of

children’s physical

ability (capacity

and performance)

(Generic)

Ages 5 to 15 years old Physical activity

assessment in

adolescents with

limb salvage (6)

Pediatric Outcomes Data

Collection Instrument

(PODCI)

Functional and

health outcomes

associated with

paediatric

orthopaedic

conditions

(Generic)

Child and parent

report for ages 11-18

years; parent proxy

report for children

ages 10 years and

under

The correlation of

specific orthopaedic

features of

polyostotic fibrous

dysplasia with

functional outcome

scores in children (4)

Parental perspectives

on leg length

discrepancy (5)

PedsQL Measure of health

related quality of

life

(Generic)

Child report for ages

5-7 years, 8-12 years,

13-18 years; parent

report for ages 2-4

years, 5-7 years, 8-12

years

Measurement of

health-related quality

of life in children

undergoing external

fixator treatment for

lower limb

deformities (2)

The following section summarizes the sample population in each study and what each study

revealed with respect to the PRO measure it utilized. The level of detail in this summary is

considered important to justify the need for this thesis study.

When the Child Health Questionnaire (CHQ) was administered by parent-proxy, in a sample of

children undergoing femoral lengthening, widespread ceiling effects were noted with many of

15

the domain scores close to the maximum scores. Accordingly, it was speculated this measure

may not be adequately sensitive to change or discriminative for the study population (3).

The administration of the Gillette Functional Assessment Questionnaire (FAQ) in children

undergoing EFT showed limited discriminative ability and inability to detect change following

EFT (2). The FAQ was originally developed for children with CP and the reliability and validity

of this assessment has not been established in children with LLAs, putting in question its use as

an outcome tool for this population (78).

Monpetit and colleagues (2) used the PedsQL and showed that it was able to discriminate

between children with leg length discrepancies (LLD) from normative data (derived from healthy

children), and was responsive to change over time as a result of EFT. On the other hand, the

PedsQL effect size related to the impact of EFT was small (total score and physical health

domain) to small-medium (psychosocial health and emotional functioning domain) for child

report, and small (total score and all domains) for parent report, as measured between baseline to

3 months post frame removal. It is possible that this might be an accurate reflection of EFT

impact, however, given the magnitude of clinical change seen with EFT (e.g., up to 20% increase

in limb length) this might also suggest the PedsQL is too generic and therefore underestimates

potentially important clinical change. Further, it remains unclear if the PedsQL can discriminate

among different severities of LLD or if severity impacts responsiveness.

Sheiko et al. (6) used the ASKp-38 for 20 children (mean age 15.8 years) who had undergone

limb salvage to treat bone sarcomas (mean time since initial procedure or major revision = 2.46

years) versus a matched cohort of typically developing youth. Their results show that the

summary score differentiated between groups (p=0.001), however this was related only to

differences in the locomotion subscale scores. The mean score of the other three domains

(personal care, standing, and transfers) was 100, the maximum score of the scale. This

corroborates a previously reported concern about the ceiling effects due to a lack of higher-level

items in the ASK (e.g., competitive sports, climbing activities) (41).

Children with LLA were included in the development of the Pediatric Outcomes Data Collection

Instrument (PODCI) (79), although when Leet et al. (4) looked at the relationship between

16

PODCI scores with specific orthopaedic features, they found no correlation between PODCI

score and deformity of the limbs. In general, the PODCI is considered difficult to score and does

not offer a ‘not applicable’ option for items that are not relevant to a respondent (41).

Lee et al. used the PODCI (5) to investigate the perspectives of parents of children, ages 6-20

years, with an LLD. They found that pain and global function subscale scores were significantly

lower (p<0.05) for children that have a LLD secondary to trauma versus idiopathic etiology.

However, they did not find a correlation between the magnitude of LLD and any of the PODCI

subscale scores, and thus concluded the PODCI may not be useful in evaluations of children with

LLDs.

Within the context of the same study, Lee et al. (5) devised a new outcome measure, the LLD

Questionnaire, with an increased focus on psychological aspects. They administered the LLD

Questionnaire to the same parent group. This outcome measure consists of 34 items generated

through literature review, a semi-structured interview with parents of children with LLDs (no

indication of sample size is provided) who were asked “What are your concerns regarding LLD

of your child?” Item consensus was achieved through a panel discussion involving three

paediatric orthopaedic surgeons, 1 orthopaedic resident, 1 nurse, and 2 research assistants. Items

are organized into six domains: parent’s satisfaction, effect of LLD, preferred treatment, parent’s

mood, concern, and expectation with regard to the treatment, and are answered using a 5-point

Likert scale. Retrospectively Lee noted, “some issues could have been addressed more

accurately by putting them directly to the patients.” The study publication indicated that the

questionnaire was available as supplementary digital content however the provided link is not

currently accessible. No further psychometric testing of this questionnaire has been published.

There are many instances in which researchers have used a non-validated outcome measure or

questionnaire they created specifically for their study with children with LLAs. Ramaker (7)

developed a study-specific questionnaire to look at the social function of children after Ilizarov

leg lengthening. This questionnaire, a separate child and parent report, contained items

regarding current function at school, activities of daily living, physical and social activities, and

treatment related experiences, rated using a 5-point Likert scale. The number of items and the

item details were not described, nor are they available. Similarly, no psychometric evaluation of

17

this questionnaire has been reported.

McCarthy (8) compared function after amputation and lengthening in children with fibular

hemimelia in a 3-item measure that evaluated activity using a 4-point Likert scale (0=no

restrictions, 1-mild restrictions with strenuous activity, 2=limitation of activity, 3= severe

limitation of activity), pain using a 5-point scale (0=no pain, 1= any pain, even after strenuous

activity, 2= mild pain, 3= moderate pain, 4= severe pain), and satisfaction on a 2-point scale

(yes/no). Das (80) later used the activity item in this measure to evaluate “total lower limb

function” in children with bilateral fibular hemimelia that had undergone angular correction. No

psychometric testing of this questionnaire has been published.

Paley et al (9) devised a functional outcome scoring system to evaluate the outcome of femoral

lengthening. The scoring details were published in the original article and are easily

reproducible. Referred to as the Paley functional criteria, clinical and radiographic criteria

(clinician measures) are used (i.e., ROM of the knee, gait, mLDFA, and amount of lengthening)

in combination with patient report (i.e., pain and ability to perform activities of daily living

(ADLs) or work). Gait was evaluated on clinical examination and rated as no limp, slight limp,

or moderate limp. Patients rated their pain as none, slight, moderate, or severe and their

participation in activities/work as full, reduced, or none. There is no published evaluation of the

psychometric properties of this scoring system. Despite this, several other researchers have used

the measure (81, 82). There is concern that using unpublished measures creates bias, illustrated

by Marshall (83) who showed in schizophrenia randomized control trials, authors were more

likely to report superior treatment results versus control when using an unpublished versus

published measure, a finding most pronounced in non-pharmacological studies.

In summary, existing PRO measures of function are limited in their discriminative ability and

ability to detect change with intervention when used with children having LLAs. Consequently,

these measures are infrequently used in LLA-related outcomes research, thus our knowledge of

outcomes at the level of activities and participation is limited in this population. The Gait

Outcomes Assessment List (GOAL) (described in 4.1) is a recently developed outcome measure

with the potential to overcome the limitations of existing PRO measures of function. The need

for a new standardized functional outcome measure for use with children with LLAs has been

identified by several researchers to improve the uniformity and comprehensiveness of patient

18

assessment, to ensure fair comparisons across intervention studies, and ultimately to provide

evidence for the assumed functional benefit of surgery (10-13). Moreover, an outcome measure

that identifies patient priorities and goals would facilitate treatment planning that addresses the

specific goals and expectations of each family. According to Varni (73) the hallmark of pediatric

comprehensive care is “multidimensional assessment leading to targeted interventions based on

patient perceived needs”.

19

Chapter 4 Gait Outcomes Assessment List (GOAL)

The GOAL is a child and parent report questionnaire that evaluates the functional aspects of gait

and mobility related activities and symptoms. The overall purpose of the original GOAL was to

evaluate the physical, psychological, and social gait-related function of ambulant children with

cerebral palsy (CP). It was designed for use with children aged 9 to 18 years and their parents.

The GOAL is a hybrid measure, combining the specificity of an individualized measure (to allow

identification of patient priorities for treatment) with the standardization of a fixed item

questionnaire. It is intended to be self-administered. The GOAL is grounded within the ICF

framework (84, 85), and was developed with a view to ultimately broadening its application to

any childhood condition that is associated with gait related problems.

4.1 Background and Rationale

The GOAL was first conceptualized as an outcome measure within the context of a randomized

control trial involving children with ambulatory CP, and was created because no existing

outcome measure fulfilled the study’s objective of evaluating function and children’s and

parents’ priorities for orthopaedic surgical intervention (84, 85).

As such, GOAL development proceeded from Narayanan’s (47) Priority Framework for

Outcome Assessment (Figure 2). At the centre of this framework is the health condition of

interest. Priorities are influenced by an individual’s concerns, desires, and expectations, as they

relate to this health condition. Goals are a reflection of one’s priorities and guide the decision-

making process related to interventions. The effectiveness of these interventions is evaluated by

outcome measures. For these outcome measures to be meaningful they must, in turn, reflect the

original goals. Moreover, the framework recognizes that multiple stakeholders are involved in

health care decision-making, and although their perspectives might overlap, they may not be

concordant.

20

Figure 2. Priority Framework for Outcomes Assessment (adapted from Narayanan, 2015)

The current version of the GOAL (3.1) consists of 50 items that assess seven domains: (A)

activities of daily living & independence, (B) gait function & mobility, (C)

pain/discomfort/fatigue, (D) physical activities/games & recreation, (E) gait appearance, (F) use

of braces and assistive devices, and (G) body image & self-esteem. Domains that examine self-

perceived ability (A, B, D, E) use a 7-point ordinal scale anchored from 0 = “extremely

difficult/impossible” to 6 = “no problem at all”. Domains that examine an individual’s feelings

(F, G) use a 5-point ordinal scale from 0 = “very unhappy” to 4 = “very happy”. Symptoms of

pain/discomfort or fatigue are rated for their frequency (C) from 0 to 5, with 0 = “every day” to 5

= “none of the time”. The questionnaire also collects information on the level of assistance

required (A), walking aid used (B), and pain intensity (C). The GOAL asks children to reflect on

their abilities and experiences in the past 4 weeks. Furthermore, the GOAL captures the

perceived importance (the priority) of improving the outcome of each of its 50 items as rated on

a 5-point ordinal scale from 0 = “not a goal” to 4 = “extremely important”. Figure 3 illustrates

the 7-point ordinal scale used in four GOAL domains that evaluate self-perceived ability.

21

Figure 3. Example Scale Used in GOAL Domains Evaluating Self-perceived Ability

Studies have shown that respondents have difficulty using scales with greater than seven

response options although the optimal number of response options is unclear (86). A greater

number of options may increase ability to measure change however if there is not a meaningful

difference between response options, the reliability of the questionnaire may be negatively

impacted. For example, while it may be ideal for the GOAL to use a consistent 7- point scale

throughout, it became problematic in its creation because of the diversity of the domains. Figure

4 illustrates the 5- point ordinal scale used in GOAL domains that examines an individual’s

feelings or emotions. The GOAL development team felt it would be difficult to convert this to a

7- point ordinal scale and maintain a meaningful difference between each option.

Figure 4. Example Scale Used in GOAL Domains Evaluating Emotions

Table 2 illustrates the categorization of items in the GOAL (84) compared to previously

identified standardized outcome measures used in pediatric LLA research (Table 1) applying the

ICF linkage rules. This shows that items in the GOAL cover the domains of the ICF framework

more broadly than any other functional outcome measure evaluated, and representation of

personal factors exceeds all other measures where most have no items pertaining to personal

factors. These same results were seen when items of the GOAL were compared to standardized

outcome measures frequently used with children with CP (84).

22

Table 2. Categorization of the Items in the GOAL and the Outcome Measures Identified in

Published Paediatric LLA-related Studies, using the ICF Framework

Measure Percentage of Concepts in Each Category

Body

Structures

Body

Functions

Activities and

Participation

Environmental

Factors

Personal

Factors

Not

Definable

GOAL1

5% 25% 43% 11% 16% 0%

CHQ2 0% 27% 55% 5% 0% 12%

FAQ1 0% 9% 91% 0% 0% 0%

ASK1

0% 5% 93% 2% 0% 0%

PODCI1

0% 15% 52% 22% 6% 5%

PedsQL 4.02

0% 33% 52% 11% 0% 4% Table 2 Legend:

1adapted from (84).

2(45)

As development of the GOAL is in progress at the time of writing this thesis, there are currently

no publications specific to it. However, elements of the developmental process, related to

children with CP, have been exhibited as poster presentations at various conferences (84, 85, 87),

and the developer (UN) and his research team have been a ready resource during completion of

this thesis. A detailed description of the development of the GOAL from inception to present

state is provided in Appendix 2. This process informed the methods and decisions taken in the

current thesis project that evaluates and progressively adapts the GOAL for use with children

with LLAs.

4.2 Application of the Gait Outcomes Assessment List to Children with Lower Limb Anomalies- Sensibility

The GOAL appears to have good potential as a comprehensive outcome measure for evaluating

physical and psychosocial gait-related function in children with LLAs, given the breadth of its

content, however, to date it has only been used for ambulant children with CP, and not all aspects

of its sensibility have been explored.

Sensibility refers to “an aggregate of properties that make up the common sense aspect of an

instrument, including face and content validity” (88). A framework for evaluating sensibility

was proposed by Feinstein (89), consisting of 21 principles grouped into seven domains: purpose

23

and framework, comprehensibility, replicability (clarity of instruction), suitability of the output

scale, face validity, content validity, and ease of usage. Sensibility assessments are increasingly

being applied in studies that evaluate the quality of a questionnaire. One of the first examples of

this evaluation is by Rowe and Oxman (88) who created a sensibility questionnaire based on the

tenets of Feinstein’s framework to assess an asthma quality of life measure. This has since been

adapted by others to assess, for example, the HIV Disability Questionnaire (HDQ) (90), and the

Evidence-Based Practice Confidence (EPIC) Scale (91). Moreover, the concept of sensibility is

embedded within several of the available frameworks that are available to guide the development

and subsequent evaluation of the methodological quality of standardized outcome measures such

as the Qualitative Assessment of Physical Activity Questionnaire (QAPAQ) checklist (88, 92),

the Consensus-based Standards for the selection of health status Measurement Instruments

(COSMIN), and the Scientific Review Criteria presented by the Medical Outcomes Trust and its

Scientific Advisory Committee (SAC) (93, 94).

Not all aspects of sensibility have been explored for the original GOAL for children with CP.

Face and content validity has been established using direct input from children with CP, their

parents, and HCPs from different disciplines who care for these children (Appendix 2).

However, aspects of comprehensibility, clarity of instructions, suitability of the output scale and

ease of usage have only been assessed by HCPs (87) but have not yet been formally assessed by

children or parents. Further, the results cannot be assumed to apply to populations other than

children with CP. Therefore, an assessment of the overall sensibility of the GOAL that is

modified for children with LLA (to be known as the GOAL-LLA) is essential prior to in-depth

psychometric evaluation. Feinstein considered this of fundamental importance, stating, “if it is

not sensible, it does not warrant all the subsequent effort used to demonstrate its [internal]

consistency, criterion, and construct validity”. Since the purpose and framework of the GOAL

has already been well established (described in 4.1), this thesis study focuses on the other six

domains of sensibility evaluation (i.e., face and content validity, comprehensibility and ease of

usage, clarity of instructions and suitability of the scale) from the perspective of key stakeholders

(i.e., children with LLAs, their parents, and HCPs who care for children with LLAs). Moreover,

the study design of this thesis project provides an ideal opportunity for the results of the

sensibility assessment to inform progressive content adaptation of the GOAL-LLA.

24

Directly capturing not only an adult (parent and HCP) perspective via GOAL-LLA feedback, but

the perspectives of these children, ages 9 years and up, is of particular interest for several

reasons. Children are beginning to participate in clinical and surgical decision-making at this

point in their development, so it is valuable to gain insight into the issues they feel are important

during this stage in their life (45, 73). Studies support this age group being capable of accurate

self-report (95). Additionally, this age range aligns well with the timing of several of the

surgical options available. Although EFT may be used at any age, it is often used in combination

with growth dependent interventions (i.e., epiphyseodesis and guided growth techniques) to

achieve lower limb symmetry by the onset of skeletal maturity. Although the exact timing of

these interventions is determined by the magnitude of discrepancy or malalignment, they are

most often performed within the last few years of a child’s predicted growth period, consistent

with the age range used in this study.

25

Chapter 5 Rationale and Study Objectives

5.1 Rationale

Given the need for a comprehensive functional outcome measure specifically for children with

LLA, the starting point for this study was the consideration of the use of the GOAL for these

children. Despite some commonality in the physical impairments that characterize CP and LLAs,

such as range of motion limitations, strength deficits, and altered gait biomechanics, the

experiences of these populations may be quite different with respect to physical function. This is

because individuals with CP may also have spasticity, upper extremity involvement, and

involuntary movements of the extremities that might further hinder function or affect it in

different ways. We also anticipated that if the GOAL was used among children with LLAs,

some of the original item content that made sense for use with children with CP might be too

easy (e.g., getting in and out of bed, standing at a sink or counter) (face and content validity

issues). Consequently, we did not expect every item of the GOAL would be considered an

important goal of treatment, meaning that some items might need to be eliminated or modified to

ensure that the questionnaire content is relevant and meaningful for outcome measurement with

children having LLAs.

Moreover, not all aspects of sensibility of the GOAL have been comprehensively explored

during its development for children with CP. We believed there was a possibility that the

language could be too advanced (comprehensibility), and the questionnaire length (ease of usage)

might be a deterrent to its adoption. Additionally, we felt it was important to ask children

themselves about the clarity of instruction and suitability of the GOAL items and response

options.

The use of cognitive interviews (96) in this evaluation process allows information to be collected

from children pertaining to each of the six domains of sensibility on which this study is focused

(i.e., face and content validity, comprehensibility, ease of usage, clarity of instruction, and

suitability of scale). Cognitive interviewing is considered a critical component of paediatric

outcome measure development (13, 96, 97). This approach evaluates children’s comprehension

of questionnaire items and instructions, and ensures that a questionnaire meets its intended

26

purpose (13). Cognitive interview methods, also referred to as cognitive pretesting (96),

cognitive testing (97), and cognitive debrief (98), evolved from social and cognitive psychology

(13) and are believed to compliment field-testing of questionnaires (96).

5.2 Purpose

Given the above considerations, the purpose of this study was to develop a GOAL module

suitable for children with a lower limb anomaly (GOAL-LLA) that could serve as an evaluative

measure of physical and psychosocial gait-related function. Development of the GOAL-LLA

would be accomplished through an iterative adaptation of the current GOAL 3.1, hereafter

referred to as the GOAL-CP (Appendix 3) given its original focus for use with children with CP.

5.2.1 Objectives

To create this new evaluative measure, the GOAL-LLA, the perspectives of children and their

parents, and healthcare professionals were considered in the research process that addressed the

primary objectives of this study:

Objective 1. Evaluate the suitability of the items of the GOAL-CP for use with children with

LLA (face and content validity), to inform the elimination, modification, or retention of each

item and addition of new items (content adaptations) for the GOAL-LLA.

Objective 2. Assess the sensibility of the GOAL-LLA, namely its comprehensibility, clarity of

instruction, suitability of the response scale, and ease of usage.

The secondary objectives of this study (using child and parent data) were to:

Objective 3. Compare the responses of children with LLAs with those of their parents, for the

GOAL-LLA item scores and importance ratings, to inform the future need for separate child- and

parent-report versions. It was hypothesized that while children’s responses would be positively

correlated with their parents’, children’s item scores would be higher than parents’ scores. This

is consistent with a previous study that compared PODCI and PedsQL scores between children

with congenital upper extremity deficiency and their parents, and showed parents tended to

underestimate their children’s physical and social function (99). Conversely, it was hypothesized

that children’s item importance ratings would be lower than their parents’ ratings, based on a

study by Narayanan that showed, in regard to adolescent idiopathic scoliosis, parents’ level of

27

concern and their number of serious concerns exceeded their children’s (100). A similar trend is

expected as both scoliosis and LLAs are childhood orthopaedic conditions that may be associated

with physical and psychosocial sequelae.

Objective 4. Assess the relationship between an individual’s item scores and their importance

ratings, for children with LLAs and parents separately, to assess the respondent’s interpretation

of the questionnaire’s instructions and rating system. It was hypothesized that high scoring items

would not be considered important to improve upon, hence there would be a negative correlation

between item scores and importance ratings for both children’s and parents’ scores.

28

Chapter 6 Methods

6.1 Study Design

The four objectives of this study were addressed over two phases, with the perspectives and

feedback of children with LLAs and their parents sought in Phase 1 (addressing objectives 1 to

4), while HCPs provided input in Phase 2 (addressing objectives 1 and 2).

Administering the questionnaire to children and parents and conducting cognitive interviews

with children informed the iterative adaptation of the GOAL module (initial version= GOAL-CP,

second version= GOAL-LLA 1.0, third version = GOAL-LLA 1.1) (Phase 1). The final iteration

of the questionnaire (GOAL-LLA 2.0) was guided by the analysis of the third administration of

the GOAL in Phase 1 (GOAL-LLA 1.1) in combination with data from an e-survey administered

to HCPs (Phase 2). The sequence of study events is illustrated in Figure 5.

Cognitive Interview

Children and Parents

Objectives 1,3,4

--------------------------------

Children

Objectives 1 & 2

GOAL-LLA 1.0

Questionnaire Administration

Cognitive Interview

Children and Parents

Objectives 1,3,4

--------------------------------

Children

Objectives 1 & 2

Questionnaire Administration

Questionnaire AdministrationChildren and Parents

Objectives 3 & 4

--------------------------------

Objectives 1 & 2

Healthcare Professionals

Objectives 1 & 2

GOAL-CP

GOAL-LLA 1.1

e-Survey

GOAL-LLA 2.0

PHASE 2

PHASE 1

Content Adaptation

Content Adaptation

Content Adaptation Content Adaptation

Figure 5. Illustration of Study Events

29

6.2 Child and Parent Perspectives (Phase 1)

6.1.1 Participants

Children from 9 to 18 years with a congenital, acquired, or developmental LLA (inclusive of

lower limb deformity, deficiency, and/or length discrepancy) were eligible to be in this study,

including children who had previous surgical intervention, as we believed they would provide a

unique perspective on the functional priorities that guide surgical decision-making. The lower

age for eligibility was established based on evidence that reading proficiency and cognitive

development affects the ability of those less than 8 years to accurately self-report (95).

Children were not eligible if they had cognitive impairments preventing comprehension of test

instructions or accurate self-report, an inability to read or write English, concurrent upper

extremity involvement, or neuromuscular co-morbidities or acute or systemic illness that may

influence physical function or day-to-day performance (e.g., CP, sarcoma, or juvenile arthritis).

Parents of eligible children were invited to participate in addition to, or independent of, their

child as their views are unique and should not be considered interchangeable (101). This reflects

a family-centred care view in which family members each have opportunity if they desire to

collectively guide healthcare decision-making.

6.1.2 Recruitment

Consecutive sampling over a period of seven months was used to recruit participants from the

Limb Reconstruction Program at a pediatric tertiary care hospital and the Prosthetic Clinic at a

pediatric rehabilitation hospital in the same city. The orthopaedic surgeon or physical therapy

(PT) practitioner in clinic informed prospective participants (child and parent) of their eligibility

for this study and, if they expressed interest to participate, they met with the study investigator

(JD) who described the study in detail, answered questions, and obtained written consent to

complete the GOAL questionnaire (Appendix 4). Additionally, children had the option to

consent to participate in a cognitive interview. A child’s capacity to consent was determined by

the investigator using parental input, in combination with guidelines developed for this study

(Appendix 5). This was in accordance with the study’s Research Ethics Board-approved

protocol (Appendices 6-8) for both this Phase and Phase 2 (below).

30

6.1.3 Data Collection

Demographic information pertaining to the child was collected from the child and their parent

using an Information Sheet (Appendix 9) created for this study. Specifically, the child’s gender,

age, diagnosis, and any previous surgery were recorded. The magnitude of their total lower limb

length discrepancy and the severity of deformity, based on the position of the mechanical axis

(55), were measured from their most recent full-length lower limb alignment radiograph. For

children currently undergoing EFT, values were obtained from the most recent radiograph prior

to the initiation of EFT. The investigator (JD) also tracked the gender of parents that

participated.

6.1.3.1 Questionnaire Administration (Objectives 1, 3, and 4)

The GOAL questionnaire was administered at the point of care. The consenting child and/or

parent completed the questionnaire simultaneously but independently in the same clinic room, in

the presence of the study investigator (JD) who was available to respond to, and keep detailed

notes of any questions that arose. The questionnaire instructed respondents to score each of its

items using 5-, 6-, and 7- point domain-specific ordinal scales (as described in 4.1), and rate its

importance using a 5- point ordinal scale (also described in 4.1). Our protocol varied slightly

from that of Narayanan et al. in that we did not use general importance ratings (Appendix 2) but

rather, respondents were instructed to consider how important a goal it was to improve for each

item. This is consistent with the GOAL-CP’s current use of itemized importance ratings. In the

event the child had an external fixator in situ, the child/parent was instructed to consider the

child’s pre-fixator status rather than use the time frame indicated in the questionnaire. Based on

our clinical experience, we believed that children undergoing EFT, particularly those in the

active lengthening phase, might have temporarily lower item scores consistent with their

impairments at the time. We wanted to capture feedback on which items are impacted by an

LLA, irrespective of EFT, and felt that inclusion of this specific cohort (who had recently made a

decision to undergo lengthy and intensive treatment) would provide valuable input as to what

items were most influential in their treatment decision-making process. The last page of the

questionnaire asked respondents to list items they felt were missing and should be added.

31

6.1.3.2 Cognitive Interviews (Objectives 1 and 2)

Children who also consented to participate in the cognitive interview did this with the same

researcher (JD) immediately after completing the questionnaire. A cognitive interview guide

(Appendix 10) was developed specifically based on the Cognitive Pretesting Model for Children

(96) (Appendix 11).

The child was asked to refer to their completed copy of the GOAL as they answered the

questions and probes outlined in the cognitive interview guide that were read to them by the

researcher. ‘Think aloud interviewing’ and ‘probing’ are the two main techniques employed in

cognitive interviewing (13). Think aloud interviewing is performed concurrent to questionnaire

completion with the respondent being encouraged to talk through their thought process as they

answer each question. The probing technique can be performed concurrently or retrospectively

with the interviewer asking questions to retrieve specific information from the respondent. Our

decision to use a probing technique immediately after the child had completed the questionnaire

was related to lower respondent burden and ease, as well as fear that using the alternative think

aloud method might affect the way children answered the questions (102).

Open-ended probes were used to test the child’s understanding of words, phrases, and concepts

in the GOAL that were deemed potentially problematic a priori by the development team (e.g.,

what do you think “symmetry” means?). Children were instructed to read the GOAL section

instructions and specific items aloud (e.g., can you read [item] #7 aloud?) and were then

prompted to rephrase in their own words (in your own words can you explain what is being asked

of you?). This method of probing helped to evaluate a child’s understanding of

instructions/questions and ensured that there was consistent interpretation among participants.

Children were also asked questions regarding: the scale format and response options, (did you

feel you were able to find your answer in the list of possible answers listed?), items that they felt

should be added (is there anything you would add to this section?) or eliminated (are there any

questions that you would get rid of completely from this questionnaire?), and their overall

impressions of the questionnaire (How do you feel about the amount of time it took you to

complete the GOAL?). Detailed notes were kept by JD from each child’s cognitive interview.

32

6.1.4 Analysis of Child and Parent Perspectives (Phase 1)

Preliminary analysis of the completed questionnaires in combination with the cognitive interview

responses and investigator notes was conducted during the recruitment phase on three occasions

approximately 8 weeks apart, a schedule that accommodated at least six clinic dates for the

study’s overall recruitment activities. The results of each analysis informed the iterative

refinement of the questionnaire that was subsequently piloted on the next group of respondents

(initial version= GOAL-CP, second version= GOAL-LLA 1.0, third version = GOAL-LLA 1.1).

The final iteration of the questionnaire (GOAL-LLA 2.0) was informed by the analysis of the

completed GOAL-LLA 1.1 questionnaires in combination with Phase 2 responses. All statistical

analyses for this Phase and Phase 2 objectives, were performed using R for Mac OS X (103).

6.1.4.1 Content Adaptation Analysis (Objective 1)

Descriptive statistics (mean, median, standard deviation [SD], range) were calculated separately

for children’s and parents’ item scores and importance ratings.

Mean item scores and importance ratings from children and parents were used to inform item

elimination and to highlight items that might need to be modified. Items were judged to perform

poorly, and would be considered for elimination, if both the child and parent mean item score

was within 0.5 of the maximum score (e.g., 5.5 to 6.0 out of 6.0) indicating these items would

have poor discriminate ability and be unresponsive to change, and if both the child and parent

mean importance rating was less than 0.5 out of 4.0 indicating these items were not a priority for

improvement in this population. We were cautious in the elimination of items, particularly with

the initial questionnaire administration so poorly performing items were examined in

combination with cognitive interview results to assess for opportunities to modify, rather than

eliminate. If the research team felt an item might perform better if it were modified, the item

would be altered and incorporated into the next iteration of the questionnaire for subsequent

piloting.

New items suggested by children and parents on the last page of the questionnaire or ideas

generated from cognitive interviews were considered by the research team (JD and UN) in terms

of redundancy and fit within the measurement concept. If deemed appropriate, these items were

incorporated as described above.

33

The results of the third administration of the GOAL (GOAL-LLA 1.1) were considered in Phase

2 in combination with HCP feedback, to inform the final iteration of the questionnaire (GOAL-

LLA 2.0).

6.1.4.2 Sensibility Evaluation (Objective 2)

Children’s detailed responses from the cognitive interviews were collated into a single document,

allowing general themes to be easily identified. The format of the cognitive interview guide

allowed issues to be easily categorized by four aspects of sensibility: comprehensibility (of

words and/or concepts), clarity of instructions, suitability of the response scale, and ease of

usage. The results of the sensibility assessment were used to further inform content adaptation in

each of these areas. All ideas and related changes to the GOAL-LLA were tracked to provide a

clear audit trail.

6.1.4.3 Child versus Parent Responses Analysis (Objective 3)

We aimed to compare and describe the relationship of the total item scores and importance rating

scores, between children with LLAs and their parents, using paired sample t-tests and Pearson’s

correlation coefficient respectively. The total score was calculated as a percentage of the total

possible score, taking into account missing items. These analyses were performed on data

obtained across all versions of the questionnaire (initial version= GOAL-CP, second version=

GOAL-LLA 1.0, third version = GOAL-LLA 1.1) as, despite iterative adaptation of the

questionnaire, we expected the underlying constructs to remain stable. Moreover, this

maximized the sample size. Statistical significance was determined by an alpha-level greater

than 0.05.

6.1.4.4 Item Scores versus Importance Ratings Analysis (Objective 4)

The relationship between an individual’s total item score and importance rating score, for

children and parents, was assessed using Pearson’s correlation coefficient. For the same reason

as stated above, analysis was performed on data obtained across all versions of the questionnaire

(initial version= GOAL-CP, second version= GOAL-LLA 1.0, third version = GOAL-LLA 1.1).

Likewise, statistical significance was determined by an alpha-level greater than 0.05.

34

6.1.5 Child and Parent Sample Size

The sample size target was set at 25 children and 25 parents (could be a child-parent dyad, or a

single child or parent). This was based on the number of participants in the initial development

of the GOAL-CP (n= 22 children) (85). However, the extended plan was that recruitment could

continue until informational saturation was achieved, (i.e., the point that the development team

[JD and UN] felt no new suggestions related to content adaptation, or novel input pertaining to

sensibility), were forthcoming. The required number of subjects estimated for the cognitive

debrief was 10 children based on similar studies that have used this process during the

development phase of a pediatric self-report measure (41, 104).

To broaden the sample of questionnaire respondents, a surgical caseload review was conducted

(by JD and UN) five months into the recruitment period. Children identified as eligible but not

having a scheduled clinic appointment within the remaining recruitment period, were mailed an

information letter, child and parent consent forms, and the questionnaire (child and parent

version), with a self-addressed return envelope. Families choosing to participate completed the

questionnaire at home and mailed it back. Children in this sample were not eligible for the

cognitive interview as this needed to be conducted in-person.

6.2 Health Care Professional Perspectives (Phase 2)

6.2.1 Participants

Orthopaedic surgeons, PTs, Nurse Practitioners, and PT Practitioners that have experience

working with children with LLAs were eligible for inclusion in this study. It was thought that

the inclusion of HCPs would enhance face and content validity by providing a cross-check, as

their broad clinical experience and knowledge of natural history might help to identify issues not

considered by families (105). Finally, HCP participation in the development stage might

increase the likelihood of their future utilization of this measurement outcome.

6.2.2 Sampling Recruitment

The international multi-disciplinary group of content expert HCPs was identified through their

local affiliation with our institutions or the membership lists of the Limb Lengthening and

Reconstruction Society (LLRS) and the British Limb Reconstruction Society (BLRS). Identified

35

HCPs received an electronic copy of the GOAL-LLA 1.1 (the resulting questionnaire from Phase

1), and an introductory letter (Appendix 12) including a link to an online survey. The letter

clearly stated that completing the survey implied consent to participate.

6.2.3 Data Collection

Prior to completing the e-survey, HCPs provided demographic information concerning their

occupation (orthopaedic surgeon, physical therapist, orthotist/prosthetist, or other) and years of

experience working with children with LLAs (< 5years, 5-<10 years, 10-<15 years, > 15 years).

6.2.3.1 e-Survey Questionnaire (Objective 1 and 2)

Consistent with the methodology employed in the development of the GOAL-CP, HCPs were

invited to review the GOAL-LLA 1.1 and complete the study e-survey that asked them, for each

item in the questionnaire: to accept; accept with modification and state the modification, or reject

and provide rationale for rejection. Additionally, HCPs were invited to list any other items they

felt should be included in the GOAL-LLA. Four questions were added at the end of the survey

to obtain further feedback on the GOAL-LLA 1.1 (i.e., what do you consider to be the strengths

of the GOAL-LLA, what do you consider to be the weaknesses of the GOAL-LLA, would you

consider use of the GOAL-LLA in your clinical practice, yes/no (please explain), would you

consider using the GOAL-LLA for research purposes, yes/no (please explain)). Their responses

provided additional insight into the sensibility of the questionnaire.

6.2.4 Analysis of Healthcare Professional Perspectives

6.2.4.1 Content Adaptation Analysis (Objective 1)

The frequency of HCP responses (Accept, Accept with Modification, and Reject) for the items in

GOAL-LLA 1.1 were collated for each item and percentages calculated. Items that received

greater than 3 out of 31 reject responses (> 10%) were considered for elimination. Conversely,

items that received at least 28 out of 31 accept responses (90%) were retained in the final version

of the GOAL (GOAL-LLA 2.0). Similar to the process in Phase 1, modification and new item

suggestions put forth in the HCP survey were considered by the development team (JD and UN)

in terms of redundancy and fit within the measurement concept and, if deemed acceptable, were

incorporated. If the development team felt uncertain about the fate of an item, they consulted the

36

results of the third administration of the GOAL (GOAL-LLA 1.1) to children and parents, to

obtain further feedback on the item’s performance.

6.2.4.2 Sensibility Evaluation (Objective 2)

HCP e-survey responses provided input on two domains of sensibility, specifically

comprehensibility and ease of usage.

6.2.4.2.1 Comprehensibility

The development team reviewed each of the suggested modifications and rejection rationales and

identified any comments that related to the comprehension of the GOAL. If the development

team believed that a suggestion would add clarity to the instructions or an item concept, improve

understanding of a word, increase its international applicability, and was consistent with the

original intent of the item, the modification was incorporated into the final version of the GOAL

(GOAL-LLA 2.0). In this way, the sensibility assessment informed content adaptation.

6.2.4.2.2 Ease of Usage

Responses to the open-ended questions regarding the strengths and weakness of the

questionnaire were collated and reviewed for common themes, including themes related to ease

of usage. The number of similar responses were summed and calculated as a percentage. The

frequency of HCP responses related to the amount of time it took to administer the GOAL

(appropriate/too long) and to their considered use of the GOAL in the future (yes/no) were

collated by question and percentages calculated.

6.2.5 Healthcare Professional Sample Size

A response rate of 30-35%, although low, was considered realistic based on the response

achieved by Narayanan and colleagues when they employed a similar protocol with CP-related

HCPs in the development of the original GOAL-CP (E. Ho, personal communication, June 26,

2013). We aimed to have at least 30-35 HCPs in our sample (of varying levels of experience,

different professional backgrounds, and with international representation), so we ensured that at

least 100 HCPs received an invitation to participate.

37

Chapter 7 Results

7.1 Child and Parent Perspectives (Phase One)

7.1.1 Participants

Forty-five participants were enrolled in Phase 1. These included 25 children (14 girls) with a

mean age of 13.7 years (range, 9.0 to 17.9), and 20 parents (16 mothers). There were 20 child-

parent pairs. Sixteen children had a congenital LLA, 5 acquired, and 4 developmental. Total

lower limb length discrepancies ranged from zero to 100 mm, and deformity ranged from none to

greater than 15 degrees malalignment. Table 3 summarizes all Phase 1 participants by the

questionnaire version they were administered (initial version= GOAL-CP, second version=

GOAL-LLA 1.0, third version = GOAL-LLA 1.1). Two child-parent dyads completed the

questionnaire (GOAL-LLA 1.1) at home, with all other questionnaires completed at the point of

care. Table 4 catalogues the demographics of the child participants. Thirteen children

participated in the cognitive interviews (8 with the GOAL-CP and 5 with the GOAL-LLA 1.0).

The development team judged informational saturation had been achieved related to new item

suggestions from questionnaire administration and cognitive interviews.

Table 3. Phase 1 Participants by Questionnaire Version Administered

Children

Parents

Questionnaire

Administered

GOAL

Completion

(n)

Mean age (SD)

Range

Cognitive

Debrief*

(n)

Mean age (SD)

Range

GOAL

Completion

(n)***

GOAL-CP 12

(7 girls/5 boys)

14.8 (2.3)

12-17

8

(5 girls/3 boys)

15.7 (2.6)

13-17

11

(9 mothers)

GOAL-LLA 1.0 5

(3 girls/2 boys)

11.8 (2.6)

9-16

5

(2 girls/3 boys)

13.2 (2.7)

10-16

3

(2 mothers)

GOAL-LLA 1.1 7

(4 girls/3 boys)

13.4 (2.4)

9-17

n/a n/a 6

(5 mothers)

Total 24**

(14 girls/10 boys)

13.7 (2.6)

9-17

13

(7 girls/6 boys)

14.6 (2.5)

10-17

20

(16 mothers) Table 3 Legend:

*Cognitive debrief sample is a subsample of the children who did the questionnaire

**Total study enrollment was 25 children. One child opted not to complete the questionnaire (GOAL-LLA 1.0) but

did participate in a cognitive debrief interview ***

Each parent completed a child-parent dyad

38

Table 4. Child Participant Demographics

Child Age

(years)

Gender Etiology Side Total Leg Length

Discrepancy (mm)

A-P Lower Limb Alignment

(Deformity Grade**)

Location of

Current EFT (if applicable)

Location of

Previous EFT (if applicable)

1 16 F Tibial pseudarthrosis (C) Left 56 Valgus (1) Tibia

2 14 F Septic hip (A) Left 31 Valgus (1) Tibia

3

16

M

Fibular hemimelia (C)

Left 79

Physiological range of variation

Tibia

Tibia

Femur

4 12 M Fibular hemimelia (C) Right 50 Physiological range of variation

5 16 F Hemihypertrophy (C) Right 36 Physiological range of variation

6

17

F

Fibular hemimelia (C)

Right 15

Valgus (2)

Tibia x 2

Femur

7 17 M Tibia vara (D) Bilateral 0 Varus (2) Tibia (Bilateral)

8 17 M Genu varum (D) Right 40 Varus (3)

9 13 F Osteosarcoma (A) Right 66 Varus (2) Tibia

10 13 F Fibular hemimelia (C) Left 86 Valgus (3)

11 16 F Fibular hemimelia (C) Right 35 Physiological range of variation

12 10 M CFD (C) Right 73 Valgus (2) Femur x 2

13 13 M Genu valgum (D) Bilateral 0 Valgus (2)

14 10 F Fibular hemimelia (C) Right 50 Physiological range of variation

15 10 F Post-traumatic growth arrest (A) Left 56 Physiological range of variation

16 15 F Congenital pseudoarthrosis (C) Right 70 Physiological range of variation Tibia

17 16 M Post-traumatic growth arrest (A) Left 60 Valgus (2) Tibia

18 9 F Tibial hemimelia (C) Right 57 Physiological range of variation Tibia

19 13 M Fibular hemimelia (C) Right 40 Valgus (2) Tibia Tibia

20 13 M Perthes (D) Left 25 Physiological range of variation

21 14 M Osteomyelitis (A) Right 99 Varus (2) Femur

22 13 F CFD (C)*

23 9 F CFD (C) Right 92 Valgus (2) Femur

24 17 M Tibial hemimelia (C) Left 100 Varus (3) Tibia x 2

25 13 F Posterior medial bow of tibia (C) Left 50 Physiological range of variation

Table 4 Legend: C=Congenital, D=Developmental, A=Acquired, * child had amputation. ** Grade 1 deformity corresponds with approximately 5-10 degree malalignment,

Grade 2 with 10-15 degree malalignment, and Grade 3 with greater than 15 degree malalignment (55)

39

7.1.2 Content Adaptation (Objective 1)

The first objective of this project was to evaluate the suitability of the items of the GOAL-CP for

use with children with LLA. The results of the questionnaire administration to children and their

parents were combined with findings and recommendations arising from the cognitive interviews

of children. These informed the iterative adaptation of the questionnaire [initial version= GOAL-

CP, second version= GOAL-LLA 1.0 (Appendix 13), third version= GOAL-LLA 1.1 (Appendix

14)], summarized in Table 5.

Table 5. Summary of Content Adaptation from the Initial to Third Iteration of the GOAL

GOAL-CP GOAL-LLA 1.0 GOAL-LLA 1.1 A) Activities of Daily Living &

Independence

Getting in and out of bed Retained Eliminated

Getting in and out of a chair or wheelchair Retained Eliminated

Standing at a sink or counter Retained Eliminated

Showering or bathing Retained Eliminated

Using the toilet Eliminated

Getting dressed Modified: Putting your pants on one leg

at a time

Eliminated

Carrying an object while walking (e.g.,

toy, doll, book, cell-phone

Modified: Carrying heavy objects while

walking (e.g., grocery bags, several

school books)

Retained

Modified: Balancing objects while

walking (e.g., a cup of hot chocolate, a

tray of food)

Retained

Opening a door manually Modified: Opening a door Retained

Picking up an object off the floor Retained Retained

Getting in and out of a vehicle (e.g.,

unmodified car, van, or bus)

Modified: Getting in and out of a vehicle

(e.g., car, van or bus) Retained

B) Gait Function & Mobility

Walking for more than 250 m (around 2

blocks or 2 football fields) Retained Retained

Getting around at school (indoors) Retained Retained

Added: Getting around in crowded

spaces (concert, special event, mall) Retained

Getting around at home Eliminated

Added: Standing in long lineups Retained

Walking for more than 15 minutes Retained Eliminated

Walking faster than usual to keep up with

others

Modified: Moving quickly if I am in a

hurry (e.g., to catch a bus, late for school)

Modified: Moving quickly when

in a hurry (e.g., to catch a bus,

late for an event)

Modified: Keeping up with my friends

while walking outdoors

Modified: Keeping up with my

friends

Stepping around or avoiding obstacles Retained Retained

Going up and down stairs Retained Retained

Going up and down slopes Retained Retained

Walking on uneven ground (rough, rocky,

sandy) Retained Retained

Walking on slippery or icy surfaces

Retained Retained

C) Pain/Discomfort/Fatigue

Pain or discomfort in the feet or ankles Retained Retained

40

Pain or discomfort in the lower legs Eliminated

Pain or discomfort in the knees Retained Retained

Pain or discomfort in the thighs or hips Retained Retained

Pain or discomfort in the back Retained Retained

Feeling tired while walking Retained Retained

Feeling tired during any other physical

activities that I usually enjoy (e.g.,

swimming, running, horseback riding, or

other)

Retained Retained

D) Physical Activities, Games, &

Recreation

Using playground equipment (ladders,

monkey bars, slides, etc.)

Modified: Climbing (e.g., ladder or

stepstool) Retained

Running Retained Modified: Running fast

Participating in gliding sports (e.g.,

skating, rollerblading, skiing,

skate/snowboarding)

Retained Retained

Riding a bike or tricycle (with or without

training wheels)

Retained Retained

Swimming Retained Retained

Participating in sports that require running

(e.g., soccer, baseball, football, track) Retained Retained

Participating in sports that require jumping

(e.g., basketball, volleyball) Retained Retained

Participating in dance or martial arts (e.g.,

karate, judo, taekwondo) Retained Modified: Participating in

gymnastics, dance or martial arts

(e.g., karate, judo, taekwondo)

E) Gait Appearance

Walking with my feet flat on the ground Retained Retained

Walking taller or more upright (less

crouched or bent at the knees) Retained Retained

Walking with my feet pointing straight

ahead Retained Retained

Added: Walking without a limp

Walking without dragging my feet Retained Eliminated

Walking without tripping and falling Retained Retained

Wearing my choice of footwear (e.g.,

shoes, boots, sandals)

Modified: Moved from E) to G) Retained

F) Use of Braces and Assistive Devices

Wearing braces or orthotics (e.g., AFO) Modified: Wearing braces or orthotics

(e.g., AFO, shoe lift)

Modified: A shoe lift

Modified: A brace (e.g., AFO)

Using a walking aide (e.g., walker, stick,

cane, crutches) Retained Retained

Using a wheelchair Eliminated Re-added

Added: Other assistive devices

(e.g., built-up bicycle pedal

or________)

G) Body Image & Self-Esteem

The shape and position of my legs Retained Retained

The shape and position of my feet Retained Retained

The symmetry of my legs (in length and

size) Retained Retained

Added: Wearing my choice of clothing

(e.g., shorts, skirts, bathing suits) Retained

The appearance of how I get around

compared with others Retained Retained

The way others feel about how I get

around Retained Retained

How I am treated by others Retained Retained

The descriptive statistics (mean, SD, range) for children’s and parents’ item scores and

importance ratings for the initial and second administration of the questionnaire are presented in

41

Tables 6 and 7, respectively. The descriptive statistics for the third administration of the

questionnaire (GOAL-LLA 1.1) are presented in Appendix 15 and were considered in

combination with HCP perspectives (Phase 2) to develop the final iteration of the questionnaire

(GOAL-LLA 2.0). The details on the results of the item elimination and modification process

from Phase 1 are as follows.

7.1.2.1 Item Elimination

A total of 10 items were eliminated during Phase 1 (Table 5). Four items were eliminated after

the first administration of the GOAL (GOAL-CP) (Table 6), one item [using the toilet (A)], due

to poor rating performance (as defined in Section 6.1.4.1) and two items [getting around at home

(B), pain or discomfort in the lower legs (C)] due to the development team’s decision there was

overlap with other items. For example, getting around at home was judged to be an activity that

was not much different from getting around at school (indoors), and therefore was eliminated.

Using a wheelchair was initially eliminated as the development team felt it was rarely relevant to

the LLA population, however this item was re-added in GOAL-LLA 1.1 based on feedback from

children in cognitive interviews.

Table 6. Results of the Initial Administration of the GOAL (GOAL-CP) in Phase 1 (Children

and Parent Perspectives)

Children (n = 12) Parents (n = 11)

Domain and Items

Item Score

Mean (SD)

Range

Importance Rating

Mean (SD)

Range

Item Score

Mean (SD)

Range

Importance Rating

Mean (SD)

Range

A) Activities of Daily Living &

Independence

(/6) (/4) (/6) (/4)

Getting in and out of bed 5.9 (0.3)

5-6

0.6 (1.0)

0-2

5.3 (1.3)

3-6

1.8 (2.2)

0-4

Getting in and out of a chair or

wheelchair

5.8 (0.6)

4-6

0.4 (0.8)

0-2

5.3 (1.3)

3-6

1.8 (2.2)

0-4

Standing at a sink or counter 5.6 (0.8)

4-6

0.4 (0.7)

0-2

5.5 (0.9)

4-6

1.8 (2.2)

0-4

Showering or bathing 5.7 (0.9)

3-6

0.8 (1.5)

0-4

5.4 (1.1)

3-6

1.9 (2.3)

0-4

Using the toilet * 5.9 (0.3)

5-6

0.4 (1.3)

0-4

5.5 (0.9)

4-6

1.9 (2.2)

0-4

Getting dressed 5.8 (0.6)

4-6

0.6 (1.3)

0-4

5.5 (0.9)

4-6

1.8 (2.2)

0-4

Carrying an object while walking (e.g.,

toy, doll, book, cell-phone

5.7 (0.6)

4-6

0.3 (0.7)

0-2

5.1 (1.4)

2-6

2.3 (2.2)

0-4

Opening a door manually 5.8 (0.6)

4-6

0.2 (0.4)

0-1

5.4 (0.9)

4-6

2.3 (2.0)

0-4

Picking up an object off the floor 5.6 (1.0)

3-6

0.4 (1.0)

0-3

4.5 (1.6)

2-6

2.2 (2.2)

0-4

Getting in and out of a vehicle (e.g.,

unmodified car, van, or bus)

5.8 (0.6)

4-6

0.6 (1.1)

0-3

5.3 (1.1)

3-6

2.0 (2.1)

0-4

42

B) Gait Function & Mobility (/6) (/4) (/6) (/4)

Walking for more than 250 m (around 2

blocks or 2 football fields)

4.6 (1.7)

1-6

0.7 (1.3)

0-3

3.5 (1.8)

1-6

2.7 (1.8)

0-4

Getting around at school (indoors) 5.5 (1.0)

3-6

0.2 (0.6)

0-2

5.0 (1.1)

3-6

1.7 (2.1)

0-4

Getting around at home * 5.7 (0.8)

4-6

0.2 (0.6)

0-2

5.3 (0.9)

4-6

1.7 (2.1)

0-4

Walking for more than 15 minutes 4.7 (1.5)

1-6

0.7 (1.1)

0-3

3.7 (2.2)

1-6

2.7 (1.8)

0-4

Walking faster than usual to keep up

with others

4.5 (1.5)

2-6

1.1 (1.3)

0-3

3.4 (1.7)

0-6

2.4 (1.8)

0-4

Stepping around or avoiding obstacles 5.4 (1.0)

3-6

0.2 (0.6)

0-2

4.7 (1.1)

3-6

2.2 (1.9)

0-4

Going up and down stairs 5.4 (1.2)

2-6

0.3 (0.9)

0-3

4.6 (1.4)

3-6

2.1 (2.0)

0-4

Going up and down slopes 4.3 (1.4)

2-6

0.7 (1.1)

0-3

3.4 (1.5)

1-6

2.0 (1.9)

0-4

Walking on uneven ground (rough,

rocky, sandy)

4.0 (1.8)

2-6

1.1 (1.6)

0-4

3.7 (1.4)

2-6

2.4 (1.7)

0-4

Walking on slippery or icy surfaces

4.0 (2.0)

0-6

1.0 (1.6)

0-4

3.4 (1.6)

1-6

2.9 (2.0)

0-4

C) Pain/Discomfort/Fatigue (/5) (/4) (/5) (/4)

Pain or discomfort in the feet or ankles 3.9 (1.1)

2-5

1.8 (1.3)

0-4

4.1 (0.9)

2-5

1.7 (1.7)

0-4

Pain or discomfort in the lower legs * 4.1 (0.9)

2-5

1.7 (1.6)

0-4

3.7 (0.8)

2-5

2.9 (0.9)

2-4

Pain or discomfort in the knees 3.9 (1.2)

2-5

1.8 (1.6)

0-4

4.0 (0.8)

3-5

1.7 (1.5)

0-4

Pain or discomfort in the thighs or hips 4.2 (1.3)

1-5

1.3 (1.8)

0-4

4.1 (1.2)

2-5

2.0 (1.8)

0-4

Pain or discomfort in the back 4.4 (0.9)

2-5

0.9 (1.5)

0-4

4.3 (1.2)

1-5

2.2 (1.5)

0-4

Feeling tired while walking 4.2 (0.9)

2-5

0.9 (1.4)

0-4

3.5 (1.0)

2-5

2.7 (1.2)

0-4

Feeling tired during any other physical

activities that I usually enjoy (e.g.,

swimming, running, horseback riding,

or other)

3.8 (0.7)

2-5

1.8 (1.6)

0-4

3.2 (1.7)

0-5

2.7 (1.5)

0-4

D) Physical Activities, Games, &

Recreation

(/6) (/4) (/6) (/4)

Using playground equipment (ladders,

monkey bars, slides, etc.)

5.5 (0.9)

4-6

0.6 (1.1)

0-3

4.0 (1.3)

3-6

2.0 (0.9)

1-3

Running 3.8 (1.2)

2-6

2.0 (1.4)

0-4

3.2 (1.5)

1-6

2.5 (1.3)

0-4

Participating in gliding sports (e.g.,

skating, rollerblading, skiing,

skate/snowboarding)

3.4 (1.6)

0-5

1.4 (1.1)

0-3

2.7 (1.9)

0-6

1.4 (1.4)

0-4

Riding a bike or tricycle (with or

without training wheels)

5.5 (0.9)

4-6

0.6 (1.1)

0-3

4.9 (1.5)

2-6

2.1 (1.5)

0-4

Swimming 4.8 (1.5)

3-6

0.8 (1.2)

0-3

5.1 (1.6)

2-6

1.7 (1.4)

0-4

Participating in sports that require

running (e.g., soccer, baseball, football,

track)

4.2 (1.2)

3-6

1.4 (1.6)

0-4

3.7 (1.4)

2-6

2.0 (1.2)

0-4

Participating in sports that require

jumping (e.g., basketball, volleyball)

4.7 (1.1)

3-6

1.6 (1.7)

0-4

3.7 (1.5)

2-6

1.9 (1.3)

0-4

Participating in dance or martial arts

(e.g., karate, judo, taekwondo)

4.6 (1.1)

3-6

1.1 (1.7)

0-4

3.0 (1.5)

1-6

2.3 (1.1)

1-4

E) Gait Appearance (/6) (/4) (/6) (/4)

Walking with my feet flat on the

ground

4.2 (1.7)

2-6

0.6 (0.9)

0-2

3.2 (1.8)

0-5

2.2 (1.4)

0-4

Walking taller or more upright (less 3.8 (1.6) 1.3 (1.5) 3.7 (1.6) 2.7 (1.4)

43

crouched or bent at the knees) 1-6 0-4 1-6 0-4

Walking with my feet pointing straight

ahead

3.9 (1.8)

1-6

0.7 (1.3)

0-4

2.7 (1.7)

0-5

2.9 (1.5)

0-4

Walking without dragging my feet 4.8 (1.3)

3-6

0.6 (1.1)

0-3

3.8 (2.0)

0-6

1.8 (1.6)

0-4

Walking without tripping and falling 5.3 (0.9)

4-6

0.4 (0.9)

0-2

4.7 (1.3)

3-6

2.0 (1.7)

0-4

Wearing my choice of footwear (e.g.,

shoes, boots, sandals)

4.5 (2.2)

0-6

1.1 (1.8)

0-4

3.0 (1.9)

0-6

2.2 (1.8)

0-4

F) Use of Braces and Assistive

Devices

(/4) (/4) (/4) (/4)

Wearing braces or orthotics (e.g., AFO) 1.5 (1.6)

0-4

0.8 (1.3)

0-3

1.9 (1.4)

0-4

2.5 (1.8)

0-4

Using a walking aide (e.g., walker,

stick, cane, crutches)

1.8 (0.8)

1-3

0.8 (1.0)

0-2

1.8 (0.8)

1-2

2.3 (1.5)

1-4

Using a wheelchair * 2 (0.8)

1-3

1.0 (1.1)

0-2

2.0 (0.8)

1-3

1.5 (0.7)

1-2

G) Body Image & Self-Esteem (/4) (/4) (/4) (/4)

The shape and position of my legs 1.7 (1.3)

0-4

2.1 (1.4)

0-4

1.7 (1.1)

0-3

2.9 (1.5)

0-4

The shape and position of my feet 2.1 (1.3)

0-4

1.6 (1.6)

0-4

2.1 (1.0)

1-4

2.1 (1.6)

0-4

The symmetry of my legs (in length and

size)

1.7 (1.2)

0-4

2.1 (1.7)

0-4

1.7 (1.0)

0-3

2.9 (1.8)

0-4

The appearance of how I get around

compared with others

1.8 (1.1)

0-4

1.7 (1.5)

0-4

2.2 (1.5)

0-4

1.9 (1.8)

0-4

The way others feel about how I get

around

2.3 (1.3)

0-4

0.9 (1.5)

0-4

2.6 (1.0)

1-4

1.2 (1.7)

0-4

How I am treated by others 3.3 (0.9)

2-4

1.8 (1.8)

0-4

3.1 (0.9)

1-4

1.8 (2.1)

0-4

Table 6 Legend: The four items eliminated after the first administration of the questionnaire (GOAL-CP) are

marked with an asterisk. The three shaded items had poor rating performance (both the child and parent mean item

score was within 0.5 of the maximum score or both the child and parent mean importance rating was less than 0.5

out of 4.0). Despite poor rating performance, standing at a sink or counter was retained (as standing had been

highlighted in cognitive interviews as a problematic activity) and getting dressed was modified for the GOAL-LLA

1.0 to make the task more difficult (putting your pants on one leg at a time). Both were ultimately eliminated due to

continued poor rating performance.

A further seven items were eliminated after the second administration of the GOAL (GOAL-

LLA 1.0) (Table 5), of which six were due to poor item performance [getting in and out of bed

(A), getting in and out of a chair (or wheelchair) (A), standing at a sink or counter (A),

washing/bathing (A), putting your pants on one leg at a time while standing (A) and walking

without dragging my feet (E)] (Table 7). Walking for more than 15 minutes was eliminated

based on children having difficulty in the cognitive debrief distinguishing this item from walking

for more than 250 meters (around 2 blocks or 2 football fields). The latter was retained because

children were able conceptualize this item in the cognitive debrief (“from swimming”, “from

track and field”, “the example is good, it makes it clear”) and also reported lower mean item

scores (i.e., more difficult) for this activity.

44

Table 7. Results of the Second Administration of the GOAL (GOAL-LLA 1.0) in Phase 1 (Child and

Parent Perspectives) Children (n = 5) Parents (n=3)

Domain and Items

Item Score

Mean (SD)

Range

Importance Rating

Mean (SD)

Range

Item Score

Mean (SD)

range

Importance Rating

Mean (SD)

Range

A) Activities of Daily Living & Independence (/6) (/4) (/6) (/4)

Getting in and out of bed * 5.8 (0.5)

5-6 0

All 0 6.0

All 6 0

All 0

Getting in and out of a chair (or wheelchair) * 5.4 (0.9)

4-6 0

All 0 6.0

All 6 0

All 0

Standing at a sink or counter * 5.6 (0.9)

4-6 0

All 0 6.0

All 6 0

All 0

Washing/bathing myself (e.g., shower or tub) * 5.8 (1.1)

5-6 0

All 0 6.0

All 6 0

All 0

Putting your pants on one leg at a time while

standing *

5.6 (0.5)

5-6 0

All 0

5.7 (0.6)

5-6 0

All 0

Carrying heavy objects while walking (e.g.,

grocery bags, school books)

5.0 (1.1)

3-6

0.5 (1.0)

0-2 6.0

All 6

0 All 0

Balancing objects while walking (e.g. a cup of

hot chocolate, a tray of food)

5.2 (1.0)

4-6

0.8 (1.0)

0-2 6.0

All 6 0

All 0

Opening a door 5.8 (0.7)

5-6 0

All 0 6.0

All 6 0

All 0

Picking up an object off the floor 5.8 (1.1)

5-6 0

All 0 6.0

All 6 0

All 0

Getting in and out of a vehicle (e.g., car, van or

bus)

5.4 (0.9)

4-6 0

All 0 6.0

All 6 0

All 0

B) Gait Function & Mobility (/6) (/4) (/6) (/4)

Walking for more than 250 meters (around 2

blocks or 2 football fields)

4.6 (1.8)

3-6

1.0 (1.4)

0-3

5.3 (1.2)

4-6

1.5 (2.1)

0-3

Getting around at school (indoors) 5.8 (1.1)

5-6 0

All 0 6.0

All 6 0

All 0

Getting around in crowded spaces (e.g. a

concert or special event, the mall)

5.4 (0.9)

5-6 0

All 0 6.0

All 6 0

All 0

Standing in long lineups 4.8 (1.6)

3-6

0.3 (0.5)

0-1

5.7 (0.6)

5-6 0

All 0

Walking for more than 15 minutes 5.0 (1.6)

3-6

0.5 (1.0)

0-2

4.3 (1.5)

3-6

3.5 (0.7)

3-4

Keeping up with my friends while walking

outdoors

4.8 (1.6)

3-6

0.5 (1.0)

0-2

4.3 (2.1)

2-6

2.0 (2.8)

0-4

Moving quickly if I am in a hurry (e.g., to catch

a bus, late for school)

5.0 (1.6)

3-6

0.5 (1.0)

0-2

5.7 (0.6)

5-6 0

All 0

Stepping around or avoiding obstacles 5.2 (1.5)

4-6

0.3 (0.5)

0-1

5.7 (0.6)

5-6 0

All 0

Going up and down stairs 5.6 (1.4)

5-6 0

All 0

5.7 (0.6)

5-6 0

All 0

Going up and down slopes 5.0 (2.0)

4-6

0.3 (0.5)

0-1

5.7 (0.6)

5-6 0

All 0

Walking on uneven ground (rough, rocky,

sandy)

5.2 (1.5)

3-6 0

All 0

5.3 (0.6)

5-6

1.5 (2.1)

0-3

Walking on slippery surfaces (wet or icy) 4.6 (1.5)

3-6

0.3 (0.5)

0-1

5.3 (0.6)

5-6

1.5 (2.1)

0-3

C) Pain/Discomfort/Fatigue (/5) (/4) (/5) (/4)

Pain or discomfort in my feet or ankles 3.8 (1.3)

2-5

1.3 (1.3)

0-3

3.7 (1.2)

3-5

2.0 (2.8)

0-4

Pain or discomfort in my knees 4.4 (1.5)

3-5

0.6 (0.6)

0-1

4.3 (0.6)

4-5

3.5 (0.7)

3-4

Pain or discomfort in my thighs or hips 4.6 (1.1)

4-5

0.5 (0.5)

0-1

4.0 (1.0)

3-5

3.5 (0.7)

3-4

Pain or discomfort in my back 5.0 (1.1)

All 5 0

All 0

4.7 (0.6)

4-5

3.5 (0.7)

3-4

Feeling tired while walking 3.0 (2.0)

0-5

1.5 (1.9)

0-4

4.0 (1.7)

2-5

2.0 (2.8)

0-4

45

Feeling tired during any other physical

activities that I usually enjoy (e.g., swimming,

running, horseback riding or other sport)

3.4 (1.8)

1-5

1.8 (1.5)

0-3

4.0 (1.0)

3-5

3.5 (0.7)

3-4

D) Physical Activities, Games, & Recreation (/6) (/4) (/6) (/4)

Running 3.4 (1.8)

1-6

1.8 (1.3)

0-3

4.0 (1.7)

3-6

3.5 (0.7)

3-4

Participating in gliding sports (e.g., skating,

rollerblading, skiing, skate/snowboarding)

3.0 (1.7)

2-4

0.7 (0.6)

0-1

3.3 (2.3)

2-6

2.5 (0.7)

2-3

Riding a bicycle 4.5 (1.2)

3-6

0.7 (0.6)

0-1

4.0 (1.7)

3-6

3.5 (0.7)

3-4

Swimming 5.4 (1.2)

4-6

0.5 (1.0)

0-2

5.7 (0.6)

5-6

1.0 (1.4)

0-2

Participating in sports that require running (e.g.,

soccer, baseball, football, track)

4.5 (1.0)

4-5

0.3 (0.5)

0-1

3.3 (2.5)

1-6

2.5 (0.7)

2-3

Participating in sports that require jumping

(e.g., basketball, volleyball)

4.8 (1.5)

2-6

0.3 (0.5)

0-2

3.7 (2.1)

2-6

2.5 (0.7)

2-3

Participating in dance or martial arts (e.g.,

karate, judo, taekwondo)

4.8 (1.3)

3-6

1.3 (1.5)

0-3

4.0 (2.8)

2-6

2.0 (1.4)

1-3

Climbing (e.g., ladder or stepstool) 5.3 (1.7)

3-6 0

All 0

5.7 (0.6)

5-6 0

All 0

E) Gait Appearance (/6) (/4) (/6) (/4)

Walking with my feet flat on the ground 4.6 (2.2)

1-6

1.0 (2.0)

0-4

4.7 (1.2)

4-6

1.5 (2.1)

0-3

Walking taller or more upright (less crouched

or bent at the knees)

5.0 (2.3)

1-6

1.0 (2.0)

0-4

5.7 (0.6)

5-6 0

All 0

Walking with my feet pointing straight ahead 4.4 (1.8)

1-6

1.0 (2.0)

0-4

5.3 (1.2)

4-6

1.0 (1.4)

0-2

Walking without dragging my feet * 5.6 (1.8)

1-6

1.0 (2.0)

0-4 6.0

All 6 0

All 0

Walking without tripping and falling 5.0 (2.2)

3-6

0.5 (1.0)

0-2

5.7 (0.5)

5-6

2.0 (2.8)

0-4

Walking straight and level (less leaning to the

side)

4.2 (2.7)

0-6

1.0 (2.0)

0-4

5.0 (1.7)

3-6

1.5 (2.1)

0-3

F) Use of Braces and Assistive Devices (/4) (/4) (/4) (/4)

Wearing braces or orthotics (e.g., shoelift,

AFO)

2.5 (1.5)

0-4

1.7 (2.1)

0-4

3.3 (1.2)

2-4

1.5 (2.1)

0-3

Using a walking aide (e.g., walker, stick, cane,

crutches)

4.0 (1.3)

1-4

1.0 (0)

All 1.0

n/a n/a

G) Body Image & Self-Esteem (/4) (/4) (/4) (/4)

The shape and position of my legs 2.4 (1.6)

1-4

2.0 (2.3)

0-4

2.3 (2.1)

2-4 0

All 0

The shape and position of my feet 2.6 (1.7)

0-4

1.5 (1.9)

0-4

3.0 (1.0)

1-4

1.0 (1.4)

0-2

The symmetry of my legs (in length and size) 2.6 (1.4)

1-4

2.0 (1.6)

2-5

3.0 (1.0)

2-4

2.5 (0.7)

2-3

Wearing my choice of footwear (e.g., shoes,

boots, sandals)

3.2 (1.5)

2-4

0.8 (1.0)

0-2

3.3 (1.2)

2-4

1.0 (1.4)

0-2

Wearing my choice of clothing (e.g., shorts,

skirts, bathing suits)

2.2 (1.6)

0-4

2.0 (1.8)

0-4

3.0 (1.0)

2-4

1.0 (1.4)

0-2

The appearance of how I get around compared

with others

2.6 (1.6)

0-4

1.5 (1.9)

0-4

3.0 (1.0)

1-4

1.0 (1.4)

0-2

The way others feel about how I get around 2.8 (1.6)

0-4

1.3 (1.9)

0-4

3.3 (1.2)

2-4

1.0 (1.4)

0-2

How I am treated by others 3.0 (1.7)

0-4

1.3 (1.9)

0-4

3.3 (1.2)

2-4

1.0 (1.4)

0-2

Table 7 Legend: The five items eliminated after the second administration of the questionnaire (GOAL-LLA 1.0)

are marked with an asterisk. Shaded items had poor performance (both the child and parent mean item score was

within 0.5 of the maximum score or both the child and parent mean importance rating was less than 0.5 out of 4.0).

Of those items shaded but not eliminated, opening a door, picking up an object off the floor, and getting in and out

of a vehicle continued to perform poorly and were eliminated after the third administration of the questionnaire

(GOAL-LLA 1.1). Conversely, stepping around or avoiding obstacles, going up and down stairs, and pain or

discomfort in my back were retained in Phase 2 as there was no issue with their subsequent performance (Appendix

46

15). Getting around at school (indoors), getting around in crowded spaces, standing in long lineups, going up and

down slopes, and climbing were modified in Phase 2 (Table 9).

7.1.2.2 Item Modification and Retention

After the initial administration of the GOAL (GOAL-CP) all 10 items in domain A) Activities of

Daily Living & Independence, had very high mean item scores for children (greater than 5.5 out

of 6) (Table 6). In other words, children felt they could complete these activities with great ease.

However, the development team felt several of these items were of potential content value if they

were modified to increase their level of difficulty. For example, based on feedback from

children’s cognitive interviews, the item carrying objects while walking (e.g., toy, doll, book,

cellphone) (A) was adapted and made into two items, carrying heavy objects while walking (e.g.,

grocery bags, school books) and balancing objects while walking (e.g., a cup of hot chocolate, a

tray of food). Each of these items was retained through all subsequent iterations of the GOAL-

LLA.

Wearing my choice of footwear was initially in E) Gait Appearance which instructs the child to

“rate how much of a problem you experienced with… ”. The development team believed it was

more important to capture children’s feelings about their ability/inability to wear their preferred

footwear than about the perceived difficulty of using footwear itself. Thus, the item was moved

to domain G) Body Image & Self Esteem, piloted in the GOAL-LLA 1.0, and subsequently

retained in G) which instructs the child to “rate how you felt about… ”.

At the end of Phase 1, thirty of the 50 items in the GOAL-CP (60%) were retained within their

original domain and without modification (Table 5).

7.1.2.3 Item Addition

There were 5 new items added in Phase 1 based on children’s and parents’ suggestions [getting

around in crowded spaces (B), standing in long lineups (B), walking without a limp (E), use of

other assistive devices (e.g., built up bicycle pedal) (F), and wearing my choice of clothing (e.g.,

shorts, skirts, bathing, suits (G)] (Table 5). All of these were retained into Phase 2.

47

7.1.3 Sensibility Evaluation (Objective 2)

7.1.3.1 Comprehension

No participant asked for assistance or clarification in regard to language during questionnaire

administration. Overall, during cognitive interviews children demonstrated that they were able

to read and understand the words, phrases, and concepts in the questionnaire that the

development team had considered potentially problematic a priori. For example, during cognitive

interviews children were asked, “what does symmetry mean to you?” Their responses, (e.g.,

“comparable on both sides”, “split and have two equal parts”, “line down the middle”, “same

on both sides), assured us that symmetry is a word they know and a concept they understand.

Children did however, experience difficulty defining the word gait. Five of 13 children admitted

in cognitive interviews that they were not familiar with the word or were uncertain of its

meaning. Four other children thought they understood the word but provided a definition for

gate. The word has been retained as it is part of the formal title of the original questionnaire, but

the development team ensured that gait did not appear in any of the instructions or item content

of the GOAL.

7.1.3.2 Clarity of Instruction

Instructions with regard to importance ratings proved problematic in the initial administration of

the GOAL. The intended purpose of the importance ratings was to capture how important a goal

it was to improve each specific item, allowing us to identify the items that contribute most to

treatment priorities and related decision-making. Contrary to our intent, many respondents had a

more generalized interpretation of importance, as observed by the investigator (JD) during

questionnaire administration and then confirmed during cognitive interviews. For example, most

individuals believed that the ability to get out of bed independently was very important, therefore

would rate it high. However, if that individual had no difficulty with this task then it would not

be expected to be important to improve, and ought to be rated as a low importance goal within

the questionnaire. In an attempt to improve the clarity of the instructions, they were modified in

GOAL-LLA 1.1 from select how important a goal it is for you to improve each of the following

to select how important a goal it is for you to improve each of the following activities. Despite

this effort to clarify these instructions, inconsistent interpretation continued to be observed by the

investigator, specifically for parents (JD).

48

Finally, five of the 13 cognitive interview participants admitted they had not actually read the

instructions when initially completing the questionnaire.

7.1.3.3 Suitability of the Response Scale

It was evident during the first round of GOAL administration that the recall period in the GOAL-

CP (in the past 4 weeks) was problematic in D) Physical Activities, Games & Recreation, as

some of these items were seasonal [e.g., riding a bicycle (D), swimming (D)]. Given that our

questionnaire administration was primarily over the winter, several items had respondents

checking the option I did not do this in the past 4 weeks. One child commented “I’ve been living

with this a long time, I know how these activities affect me even if I haven’t done them in the past

4 weeks”. To facilitate data collection for item analysis, respondents to the GOAL-CP were

instructed to consider the past year for the activities in D). This change was then formalized in

the next iteration of the questionnaire (GOAL-LLA 1.0).

Eleven of 13 children indicated during the cognitive debrief that they were able to find their

answer in the response options provided. One child believed their difficulty identifying an

appropriate response option was item specific, reporting that he had the greatest difficulty

categorizing his pain/discomfort. There was no consensus on the number of response options

that should be used. Seven children indicated the current number of options was adequate (“it’s

great, people need options”), four felt there were too many options (in the domains that used the

7- point ordinal scale) (“there’s a LOT of options”), and two believed that there should be more

options (“like a scale of 1 to 10”). No child reported concern using a scale that varied by

domain in wording or response option numbers.

When probed, some children had difficulty distinguishing between the qualifiers (extremely

difficult versus very difficult) used with the 7- point ordinal scale (“same as very difficult”,

“there’s not much difference between very difficult and extremely difficult”). Moreover,

although the scale used an odd number of response options, the midpoint was identified as

“slightly difficult”, negating the neutrality of a natural midpoint. Based on this and the

children’s cognitive interview comments, the development team (JD and UN) decided to modify

the scale of the GOAL-LLA 1.1 by using qualifiers only as anchors for the highest and lowest

score, Figure 6. No direct feedback was obtained from children or parents on this revised scale.

49

Figure 6. Modified Scale used in GOAL Domains Pertaining to Self-perceived Ability

(implemented in the GOAL-LLA 1.1)

7.1.3.4 Ease of usage

When asked in cognitive interviews “How do you feel about the amount of time it took to

complete the GOAL” three of 13 children (23%) indicated they felt it was too long. Time

required for children to complete the questionnaire varied from 12 to 19 minutes. When asked

“How easy or difficult did you find the GOAL to answer?” nine children (69%) reported that it

was easy. Two of these children indicated that they found importance ratings more difficult than

item scores to complete, and one child stated that they had difficulty providing responses for the

items in G) Body Image & Self-Esteem.

7.1.4 Children versus Parent Response (Objective 3)

Twenty pairs of children and their parents completed the GOAL questionnaires. Children’s item

scores were moderately correlated (r=0.42) with their parents’, although this did not reach

statistical significance (p=0.07, 95%CI=-0.03 to 0.73). As hypothesized, children’s item scores

were significantly higher (p<0.05) than their parents’. Figure 7 illustrates the relationship

between children’s and parents’ item scores across each item of the GOAL-LLA 1.1.

Figure 7. Children's and Parents' Mean Item Scores for the GOAL-LLA 1.1

50

Children’s importance ratings were strongly correlated (r=0.61, p<0.05, 95%CI=0.16 to 0.85)

with their parents’ ratings. However, as hypothesized, parents’ importance ratings were

significantly higher (p<0.001) than their children’s. Figure 8 illustrates the relationship between

children’s and parents’ importance rating scores across each item of the GOAL-LLA 1.1.

Figure 8. Children’s and Parents’ Mean Importance Ratings for the GOAL-LLA 1.1

FIGURE 7 and 8 Legend: Results from the third administration of the questionnaire (GOAL-LLA 1.1) (n=6 child-

parent dyads). Items 1-6 = A) Activities of Daily Living & Independence, Items 7-17 = B) Gait Function &

Mobility, Items 18-23 = C) Pain/Discomfort/Fatigue, Items 24-31 = D) Physical Activities, Games & Recreation,

Items 32-36= E) Gait Appearance, Items 37-41 = F) Use of Braces and Assistive Devices, Items 42-49 = G) Body

Image & Self-Esteem

7.1.5 Item Scores versus Importance Ratings (Objective 4)

There was a strong inverse association between children’s (n=24) total item score and

importance rating score (r=-0.69, p<0.001, 95% CI=-0.87 to -0.37). This relationship, presented

as mean scores for the GOAL-LLA 1.1, is illustrated by comparing Figures 7 and 8.

The association between parent’s (n= 19) item scores and importance ratings (r=-0.07, p=0.81,

95% CI=-0.54 to 0.44) was insubstantial (106) and did not reach significance.

51

7.2 Healthcare Professional Perspectives (Phase Two)

7.2.1 Participants

Phase 2 included 31 respondents who completed the survey, of whom 81% were orthopaedic

surgeons, 81% had five or more years of experience working with children with LLAs, and 52%

were practicing in North America (Table 8).

Table 8. Demographics of Phase 2 Respondents (n=31)

Demographic n (out of 31)

Profession

Ortho surgeon 25

Physical Therapist 4

Other: Physical Therapy Practitioner, Nurse Practitioner 2

Experience working with children with LLAs (years)

< 5 6

5 to<10 9

10-<15 5

>15 11

Country

Canada 5

United States 11

United Kingdom 10

Europe 3

Unknown 2

7.2.2 Content Adaptation (Objective 1)

The results of the e-survey administration, in combination with the Phase 1 results of the third

administration of the GOAL (GOAL-LLA 1.1), further informed the iterative refinement of the

questionnaire [third version= GOAL-LLA 1.1, final version= GOAL-LLA 2.0 (Appendix 16)]

(Table 9).

Table 9. Summary of Content Adaptation from the Third to Final

Iteration of the GOAL

GOAL-LLA 1.1 GOAL-LLA 2.0 A) Activities of Daily Living & Independence

Carrying heavy objects while walking (e.g., grocery

bags, several school books) Modified

Moved from A) to B)

Balancing objects while walking (e.g., a cup of hot

chocolate, a tray of food) Modified

Moved from A) to B)

Opening a door Eliminated

Picking up an object off the floor Eliminated

Getting in and out of a vehicle (e.g., car, van or bus) Eliminated

B) Gait Function & Mobility

Walking for more than 250 m (around 2 blocks or 2

football fields)

Modified: Walking for more

than 250 m/ 820 feet (around

2 blocks or 2 football fields)

Getting around at school (indoors) Modified: Getting around in

52

crowded spaces (school,

concert, shopping centre)

Getting around in crowded spaces (concert, special

event, mall)

See above

Standing in long lineups Modified: Standing for a long

time (lineups/ queues, parade,

concert)

Moving quickly when in a hurry (e.g. to catch a bus,

late for an event) Eliminated

Keeping up with my friends Modified: Keeping up with

my friends while walking

outdoors

Stepping around or avoiding obstacles Retained

Going up and down stairs Retained

Going up and down slopes Modified: Going up and

down ramps/ hills

Walking on uneven ground (rough, rocky, sandy) Retained

Walking on slippery or icy surfaces Modified: Walking on wet,

slippery or icy surfaces

C) Pain/Discomfort/Fatigue

Pain or discomfort in the feet or ankles Retained

Added: Pain or discomfort in

the lower legs (shin or calf)

Pain or discomfort in the knees Retained

Pain or discomfort in the thighs or hips Retained

Pain or discomfort in the back Retained

Feeling tired while walking Retained

Feeling tired during any other physical activities that I

usually enjoy (e.g., swimming, running, horseback

riding, or other)

Modified: Feeling easily tired

during physical activities that

I enjoy (swimming, running,

or other sports)

D) Physical Activities, Games, & Recreation

Climbing (e.g., ladder or stepstool) Modified: Climbing (e.g.,

ladder, playground

equipment, climbing wall)

Running fast Retained

Participating in gliding sports (e.g., skating,

rollerblading, skiing, skate/snowboarding) Retained

Riding a bike or tricycle (with or without training

wheels) Retained

Swimming Retained Participating in sports that require running (e.g., soccer,

baseball, football, track) Retained

Participating in sports that require jumping (e.g.,

basketball, volleyball) Retained

Participating in gymnastics, dance or martial arts (e.g.,

karate, judo, taekwondo)

Modified: Participating in

sports that require balance

(e.g., gymnastics, dance,

martial arts)

E) Gait Appearance

Walking with my feet flat on the ground Retained

Walking taller or more upright (less crouched or bent at

the knees) Retained

Walking with my feet pointing straight ahead Retained

Walking without a limp Retained

Walking without tripping and falling Retained

F) Use of Braces and Assistive Devices

A shoelift Retained

A brace (e.g., AFO) Retained

Added: A prosthesis

Using a walking aide (e.g., walker, stick, cane,

crutches) Retained

Using a wheelchair Retained

53

Other assistive devices (e.g., built-up bicycle pedal

or________) Retained

G) Body Image & Self-Esteem

The shape and position of my legs Retained

The shape and position of my feet Retained

The symmetry of my legs (in length and size) Retained

Wearing my choice of footwear (e.g., shoes, boots,

sandals) Retained

Wearing my choice of clothing (e.g., shorts, skirts,

bathing suits) Retained

The appearance of how I get around compared with

others Retained

The way others feel about how I get around Retained How I am treated by others Retained

The frequency of HCP responses (Accept, Accept with Modification, and Reject), by item, is

illustrated in Table 10.

7.2.2.1 Item Elimination

Four items that were in GOAL-LLA 1.1 were eliminated in the final iteration of the GOAL

(GOAL-LLA 2.0) (Table 9). One item [opening a door (A)] received four HCP reject responses

(Table 10) too many to be retained (as previously described in 6.2.4.1). Based on concerns put

forth by HCPs related to perceived redundancy in the GOAL (described in 7.2.3.2), the

development team (JD and UN) judged that moving quickly when in a hurry (B) overlapped with

running fast (D) since both items were speed related. The latter was retained based on children

and parent’s having an overall lower item score and a wider range of item scores noted for

GOAL-LLA 1.1 (Appendix 15). The other two items eliminated were informed by Phase 1

results and were discussed in 7.1.2.1.

Table 10. Results of the Administration of the GOAL-LLA 1.1 in Phase Two (Health Care

Professional Perspectives) (n=31)

‘Accept’

(%)

‘Accept with

Modification’

(%)

‘Reject’

(%) A) Activities of Daily Living & Independence

Carrying heavy objects while walking (e.g. grocery

bags, several school books)

24 (77) 5 (16) 2 (6)

Balancing objects while walking (e.g., a cup of hot

chocolate, a tray of food)

28 (90) 1 (3) 2 (6)

Opening a door 22 (71) 5 (16) 4 (13)

Picking up an object off the floor 26 (84) 2 (6) 3 (10)

Getting in and out of a vehicle (e.g., car, van, or bus) 27 (87) 3 (10) 0

B) Gait Function & Mobility

Walking for more than 250 m (around 2 blocks or 2

football fields)

22 (71) 9 (29) 0

Getting around at school (indoors) 27 (87) 3 (10) 1 (3)

Getting around in crowded spaces (concert, special 24 (77) 6 (19) 1 (3)

54

event, mall)

Standing in long lineups 22 (71) 6 (19) 3 (10)

Keeping up with friends 24 (77) 7 (23) 0

Moving quickly when in a hurry (catch a bus, late for

event)

27 (87) 3 (10) 1 (3)

Stepping around or avoiding obstacles 29 (94) 0 2 (6)

Going up and down stairs 25 (81) 6 (19) 0

Going up and down slopes 24 (77) 6 (19) 1 (3)

Walking on uneven ground (rough, rocky, sandy) 29 (93) 2 (6) 0

Walking on slippery or icy surfaces 27 (87) 2 (6) 2 (6)

C) Pain/Discomfort/Fatigue

Pain or discomfort in the feet or ankles 28 (90) 2 (6) 1 (3)

Pain or discomfort in the knees 27 (87) 3 (10) 1 (3)

Pain or discomfort in the thighs or hips 26 (84) 4 (13) 1 (3)

Pain or discomfort in the back 29 (94) 1 (3) 1 (3)

Feeling tired while walking 19 (61) 12 (39) 0

Feeling tired during any other physical activities that

I usually enjoy (e.g., swimming, running, horseback

riding, or other)

22 (71) 8 (26) 1 (3)

D) Physical Activities, Games, & Recreation

Running fast 25 (81) 6 (19) 0

Participating in gliding sports (e.g., skating,

rollerblading, skiing, skate/snowboarding)

25 (80) 3 (10) 3 (10)

Riding a bicycle 29 (94) 2 (6) 0

Swimming 28 (90) 2 (6) 1 (3)

Participating in sports that require running (e.g.,

soccer, baseball, football, track)

27 (87) 4 (13) 0

Participating in sports that require jumping (e.g.,

basketball, volleyball)

29 (94) 2 (6) 0

Participating in gymnastics, dance or martial arts

(e.g., karate, judo, taekwondo)

28 (90) 2 (6) 1 (3)

Climbing (e.g., ladder or stepstool) 23 (77) 6 (19) 1 (3)

E) Gait Appearance

Walking with my feet flat on the ground 29 (94) 2 (6) 0

Walking taller or more upright (less crouched or bent

at the knees)

27 (87) 4 (13) 0

Walking with my feet pointing straight ahead 27 (87) 2 (6) 2 (6)

Walking without a limp 29 (94) 2 (6) 0

Walking without tripping and falling 31 (100) 0 0

F) Use of Braces and Assistive Devices

A shoe lift 25 (81) 6 (19) 0

A brace (e.g., AFO) 25 (81) 6 (19) 0

A walking aide (e.g., walker, stick, cane, crutches) 25 (81) 6 (19) 0

A wheelchair 25 (87) 6 (19) 0

Other assistive devices (e.g., built-up bicycle pedal

or________)

27 (87) 4 (13) 0

G) Body Image & Self-Esteem

The shape and position of my legs 30 (97) 1 (3) 0

The shape and position of my feet 30 (97) 1 (3) 0

The symmetry of my legs (in length and size) 25 (81) 5 (16) 1 (3)

Wearing my choice of footwear (e.g., shoes, boots,

sandals)

30 (97) 1 (3) 0

Wearing my choice of clothing (e.g., shorts, skirts,

bathing suits)

29 (94) 2 (6) 0

The appearance of how I get around compared with

others

30 (97) 1 (3) 0

The way others feel about how I get around 29 (94) 1 (3) 1 (3)

How I am treated by others 29 (94) 0 2 (6)

Table 10 Legend: Shaded items were automatically retained based on receiving at least 28 out of 31 Accept

responses. The other items required further critical appraisal to determine their appropriateness for retention or the

need to modify or eliminate.

55

7.2.2.2 Item Modification

Eleven items were modified in Phase 2 (Table 9), including two items that were moved to a

different domain (discussed in 7.2.2.4). Five of these modifications were based on HCP

suggestions and related directly to comprehension (7.2.3.1). Other changes were made to make

items more explicit (e.g., keeping up with my friends was modified to keeping up with my friends

while walking outdoors), and were based on suggestions of HCPs in their completed surveys.

7.2.2.3 Item Addition

There were two items added in Phase 2. Pain or discomfort in the lower legs (C) had been

eliminated in Phase 1, however, based on the comment of a HCP [“add shin, (sometimes kids get

bone pain, not just joint pain”)], the development team (JD and UN) felt that this item should be

included. The other addition was to include prosthetic as a separate item in F) (Table 9).

7.2.2.4 Item Retention

Two items that were retained in the GOAL-LLA 2.0 [carrying heavy objects while walking (A)

and balancing objects while walking (A)] were relocated to domain B) Gait Function and

Mobility, where the development team believed they were better aligned in concept (Table 9).

Seventeen of the 48 items were retained based on receiving at least 28 out of 31 “accept”

responses (previously discussed in 6.3.4.1). In total, 32 of the 48 items in the GOAL-1.1 (67%)

were retained (without being moved or modified) at the end of Phase 2 (GOAL-LLA 2.0).

7.2.3 Sensibility (Objective 2)

7.2.3.1 Comprehensibility

Comments from several HCPs reflected the development team’s initial concerns related to the

language used in the GOAL (e.g., “language too technical”, “symmetry probably not

understood…”, “vocab too advanced”), however, the definitions provided by the children during

cognitive interviews, described above (7.1.3.1), assured the development team the language was

appropriate.

To improve the generalizability of the questionnaire for use outside Canada the following

modifications were suggested by HCPs and implemented by the development team: distance

56

described in metres and feet, use of the term shopping centre as opposed to mall, add queue to

the list of examples provided for the item standing for a long time.

7.2.3.2 Ease of Usage

All 25 HCPs who identified strengths of the GOAL focused on its content, with 13 (52%)

commenting specifically that the questionnaire’s level of detail (comprehensiveness) was an

asset. The majority of responses (18 of 20 HCPs) identified weaknesses of the GOAL that could

be categorized as either its length or its complexity. 12 respondents (60%) indicated the length

was a weakness, of whom 4 specifically reported that there was redundancy of items.

Fifteen of 31 HCPs (48%) responded that the amount of time it would take for the GOAL-LLA

to be administered, was “too long”. Moreover, 5 HCPs indicated they would not consider the

GOAL-LLA for clinical use related to time constraints and lack of support staff to assist with

administration. Overall, after reviewing the GOAL-LLA 1.1, 23 of 30 (77%) would consider

using it in their clinical practice and twenty-seven of 30 HCPs (90%) reported they would

consider using this questionnaire for research purposes.

7.3 GOAL-LLA 2.0

The final iteration of the GOAL questionnaire, reflecting the cumulative results of Phase 1 and 2

of this study, is the GOAL-LLA 2.0 (Appendix 16). This questionnaire has 44 items organized

into six domains. Twenty-two items from the original 50 items (GOAL-CP) (44%) were retained

through each iteration of the questionnaire (with no modifications). Six items were new and 16

original items were modified. Domain A) Activities of Daily Living & Independence was

eliminated as a result of these adaptations.

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Chapter 8 Discussion

This study aimed to develop an evaluative measure of physical and psychosocial gait-related

function suitable for children with a lower limb anomaly (GOAL-LLA), through iterative content

adaptation and questionnaire refinement of the GOAL-CP that was developed for a different

population. This process involved field-testing and sensibility evaluation of the GOAL-CP and

the subsequent two interim versions of the GOAL-LLA (GOAL-LLA 1.0 and GOAL-LLA 1.1).

LLAs encompass a wide spectrum of disorders and clinical presentations and the research team

wanted to create a questionnaire that was relevant (specific) to the issues of this diverse

population.

The decision to adapt the GOAL-CP for children with LLAs, as opposed to creating an entirely

new measure, was made for several reasons. The GOAL-CP already appeared to be a

comprehensive functional outcome measure for gait related problems that broadly covered all

domains of the ICF framework and was focused on patient priorities (84). Moreover, the content

of the GOAL-CP was generated, in part, from review of existing outcome measures that included

three measures previously used for children with LLAs (i.e., PODCI, ASK, FAQ) (84). Further,

working from the GOAL-CP as a template, required a shorter time frame than creating a new

measure. Although creating a new measure may appear to be a more comprehensive process, the

use of cognitive interviews in our study, and the opportunity for children, their parents, and

HCPs to provide suggestions for modifications and new items, ensured the adaptations through

the various iterations of the GOAL-LLA were well informed.

The inclusion of children, parents, and HCPs in the development of the GOAL-LLA module

contributes to its face and content validity, and ensures that the measured outcomes are

meaningful to all of the key stakeholders. Administration of the original GOAL-CP to children

with LLAs and their parents revealed that some of the item content was too easy, and

consequently, not considered important to change with treatment. This was true, particularly for

items in domain A) Activities of Daily Living & Independence. We found that children with

LLAs do not generally have functional issues with basic activities of daily living and

independence in contrast to children with CP, for whom these items pose a wide range of

difficulty. Domain A) was therefore eliminated from the questionnaire because its items

58

demonstrated a ceiling effect despite modifications to make them more challenging. Retaining

such items in the GOAL-LLA would impede the GOAL’s ability to discriminate between

children or its sensitivity to demonstrate change following interventions. This was one of the

limitations of generic functional outcome measures such as the ASK (6) and PODCI (5) when

applied to this population.

Conversely, domains that performed well and required minimal content adaptation were C)

Pain/Discomfort/Fatigue and G) Body Image and Self-Esteem. It appears that the original items

in these domains are generic enough to be applicable across the spectrum of LLAs as no items

were eliminated in either domain. In general, the items in these domains were the highest

priority items as indicated by their having the highest mean importance rating scores across all

versions of the questionnaire (Tables 6 and 7). For some children, cosmetic gains may be the

primary underlying motivation for surgical intervention such as EFT (2, 107, 108). Of note,

Varni and Setoguchi (109) found that the perceived physical appearance of children having a

congenital or acquired limb deficiency was significantly influenced by social environment and

perceived competence/adequacy (peer acceptance, scholastic competence, athletic competence),

and was positively correlated with self-esteem (p<0.001).

Of the six new items added, three are not typically used for children with CP (i.e., use of a

shoelift, use of a prosthesis, use of other assistive devices [e.g., built-up bicycle pedal]). Their

addition to the GOAL-LLA increased the specificity of this questionnaire module for use with

children with LLAs. Many of the modifications made based on child and parent feedback (Phase

1) were to increase the difficulty of the item content in keeping with the higher functional

abilities of this population (e.g., carrying an object while walking [e.g., toy, doll, book, cell-

phone] was modified to carrying heavy objects while walking [e.g., grocery bags, several school

books]). Subsequent modifications, informed primarily by HCPs (Phase 2) helped to refine the

language to improve specificity of the items (keeping up with friends while walking was

modified to keeping up with friends while walking outdoors); and to better reflect variations in

English language use internationally. For example ramp replaced slope as this could be

misconstrued as ski slope, and shopping centre replaced shopping mall, as this term is not

commonly known in the UK.

59

The inclusion of a multi-national group of HCPs in Phase 2 (16/28 from North America, 12/28

from the UK and Europe) was a first step in advancing the international transferability of the

questionnaire, however the results of this study may not be entirely generalizable beyond Canada

given the sample of children and parents used in Phase 1. Further cognitive interviews

conducted on small groups of children in other countries would either confirm the

generalizability of the questionnaire internationally or identify content or language that was

problematic. The results of these cognitive interviews would inform further questionnaire

modifications, or possibly, the need for a separate country/language-specific version (or module)

of the GOAL-LLA.

The use of cognitive interviews provides insight into children’s thought process and their level of

understanding while completing the GOLA-LLA. The greatest advantage of this process is the

opportunity to dialogue with the children and ensure that the questionnaire truly resonates with

them, which is difficult to ascertain through field-testing alone. The children in this study were

willing to talk about their personal experiences related to their LLA and several new item

suggestions were generated in this manner (e.g., wearing my choice of clothing, standing for a

long time).

The length of the GOAL might present a challenge for HCPs as far as administrative burden

(ease of use). The majority of children felt the length of the questionnaire was appropriate,

indicating that there is limited burden on the respondent. Often, too high a value is placed on

brevity, with many short scales having less than adequate reliability (110). Moreover, we believe

that the comprehensiveness of this questionnaire might be jeopardized if the number of items

was further reduced. Three HCPs suggested that some of the items in D) Physical Activities,

Games, & Recreation could be combined into team versus independent sports. However, each

group of activities measure a different component of gross motor function (e.g., running,

jumping, coordination) and should be evaluated separately for purposes of meaningful and

specific goal setting, intervention, and outcome evaluation.

The language of the questionnaire (comprehensibility) was appropriate for the children

participating in our study (i.e., Canadian children from 9 to 18 years of age). Fears of language

being too advanced were reflected in HCP comments (and had been a concern of the

investigators at the outset of the study), but these were not substantiated by children’s responses

60

in cognitive interviews or their performance during questionnaire administration. However, the

clarity of the instructions was problematic with regard to importance ratings of the items.

Despite modifications to the wording of these instructions, importance ratings were not always

interpreted as importance to improve with intervention, as intended by the development team.

Some parents continued to rate all items with high importance, presumably to demonstrate that

they felt the item was important to perform, indicated by their mean importance ratings being

significantly higher than children’s responses (mean difference of total importance rating scores

between children and their parents = 28.0, p<0.001). Items that received high scores would not

ordinarily be considered important to improve upon and it was judged that at least a moderate

negative correlation between item scores and their respective importance rating indicated that

respondents were properly interpreting the questionnaire’s instructions and rating system. This

pattern was observed between children’s item and importance rating scores (r=-0.69, p<0.05) but

not parents’ scores (r=-0.07, p=0.81). This is most likely attributable to misinterpretation of the

importance scoring or because the instructions were not read. Thirty-eight percent of children

participating in cognitive interviews reported they had not read the instructions. Additionally,

the investigator administering the questionnaire might have provided more detailed instruction to

the child respondent assuming they would require more direction than their parents. As such,

standardized verbal instructions may be required to ensure interpretation of importance ratings

that is consistent with the intended meaning. Inconsistent interpretation of items or instructions

among respondents invalidates the results of a questionnaire for comparative studies.

Children had significantly higher item scores and lower importance ratings than their parents.

However, there was a moderate association between both groups’ importance rating scores (r=

0.61), indicating both children and their parents had similar ideas related to what their priorities

were, despite attributing different number values to the score. This is consistent with the Priority

Framework for Outcomes Assessment (47) that indicates the perspectives of stakeholders might

overlap but might not be concordant. This affirms that child- and parent- report versions of the

GOAL-LLA should be administered to ensure that both perspectives are captured and taken into

consideration when making health-care decisions.

Scoring of the GOAL provides a total GOAL score and six separate domain scores. Importance

ratings do not contribute to the score. They are only intended to identify those items to focus on

when goal setting for intervention. Unlike other outcome measures that have previously been

61

used in this population (e.g., ASK, FAQ), the GOAL-LLA is intended, through its design, to

capture the priorities of families related to intervention (e.g., surgical, rehabilitative) goals. The

example below illustrates how the inclusion of importance ratings, results in different items

being highlighted as a consideration for intervention goals. Very importantly, this shows that the

items that are rated highest in importance are not consistent across all respondents. Individual

children and/or their parents have individual priorities that cannot be assumed to be similar even

for children with similar diagnoses and severity of involvement. The GOAL allows for easy

identification of items that are particularly problematic to the individual and that they consider

very important to improve. Therefore the GOAL allows clinicians to identify an individual’s

treatment priorities, not all of which might be feasible, that would provide an opportunity to

guide further discussion, define/refine expectations and goals, assist with informed consent, and

facilitate appropriate clinical decision-making with regard to choice of intervention.

Furthermore, effectiveness of an intervention can be judged based on positive changes

particularly in items and domains that were identified to be most important. This aligns well with

the individualized goal approach taken with measures like the COPM and GAS in which priority

goals are identified. This is illustrated in the two following examples. In the future validation of

the GOAL-LLA, it would be helpful to consider whether the GOAL scores modified by the

importance ratings might provide a more meaningful or responsive outcome measure to evaluate

effectiveness of an intervention.

a) 11-year-old girl with a congenital femoral deficiency on the right side with nearly a nine-

centimeter length discrepancy.

Child’s Total Score: 221/246 = 89.8%

Item

Domain Item Score

Importance Rating

Wearing a brace (AFO) F) Use of Braces & Assistive Devices

1/4 4/4

Wearing my choice of footwear (e.g., shoes, boots, sandals)

G) Body Image & Self-Esteem

1/4 4/4

Wearing my choice of clothing (e.g., shorts, skirts, bathing suits)

G) Body Image & Self-Esteem

2/4 4/4

The shape and position of my feet G) Body Image & Self-Esteem

2/4 3/4

Walking taller or more upright (less crouched or bent at the knees)

E) Gait Appearance 2/6 2/4

62

b) 13-year-old boy with fibular hemimelia on the right side with nearly a five-centimeter

length discrepancy.

Child’s Total Score: 220/276 = 79.7%

Item

Domain Item Score

Importance Rating

Feeling tired during any other physical activities that I usually enjoy (e.g., swimming, running, horseback riding, or other sport)

C) Pain/Discomfort/Fatigue 2/5 3/4

Running D) Physical Activities, Games & Recreation

3/6 3/4

Feeling tired while walking C) Pain/Discomfort/Fatigue 1/5 2/4 Swimming D) Physical Activities, Games

& Recreation 4/6 2/4

The way I get around compared with others

G) Body Image & Self-Esteem 1/4 2/4

There is some potential for application of the GOAL-LLA 2.0 to individuals outside of the age

range used in our study sample (9 to 18 years). The content of the questionnaire content may be

applicable to children as young as five or six years of age, who spend all day at school and share

some physical and social experiences that overlap with those of older children. The suitability of

the GOAL-LLA 2.0 for younger children would however have to be confirmed, as there may be

age related developmental considerations for some of the item responses (e.g., riding a bicycle,

swimming, walking for more than 250 meters, standing for a long time) which may be

challenging for some five to eight year-olds (regardless of the presence of an LLA). Given that

the reliability of self-report decreases for children younger than nine years (95), the use of

parent-proxy may be most appropriate for this age group.

For children less than 5 years of age the GOAL-LLA 2.0 may serve as a template from which to

create a more developmentally appropriate evaluative outcome measure. Daltroy et al. (79)

found that while developing the PODCI, the questionnaires completed for children aged two to

five years, had more missing data for items pertaining to physical function and sports, than those

for older age groups, a likely indication these type of items are less relevant to young children.

Similarly, it is likely that the GOAL-LLA 2.0 would require revision to be suitable for an adult

population. Although several domains do appear relevant to individuals over 18 years of age

(Pain/Discomfort/Fatigue, Gait Appearance, Use of Braces/Assistive Devices, and Body Image

and Self-esteem) the items in domains specific to physical function (Gait Function and Mobility,

Physical Activities/Games/Recreation) may not all be applicable. For instance, some items are

63

activities specific to a school environment or emphasize sporting activities, which adults may

find less relevant or have less time or interest to perform. Additionally, it is possible that the

negative correlation between item scores and importance ratings is weaker than that seen with

children. For example, an adult may have great difficulty running fast (i.e., low item score) but

at this stage of their life they may place less value on this as an item to improve upon with

intervention (i.e., low importance rating). Adults with LLAs may feel there are many pertinent

items missing from the questionnaire (e.g., regarding employment opportunities, adult

relationships, parenting). The suitability of the GOAL-LLA 2.0 content to children and adults

outside of the age range used in our study sample could be further explored through cognitive

interviews or focus groups and field-testing of the questionnaire.

The development of age-appropriate modules of the GOAL-LLA 2.0 would allow the

questionnaire to be administered at different stages in an individual’s life. This may provide

clinicians and researchers with a better understanding of how individuals’ priorities might

change over time. The emergence of certain trends (i.e., domains that generally become more or

less important over time) could provide children with LLAs and/or their parents with meaningful

insight into possible future issues.

8.1 Limitations

The population of children with LLAs is very diverse with a range of clinical presentations and

severity. Our sample of only 25 children included a wide range of ages (the full range of targeted

ages was sampled), both genders (56% were female), and a broad range of diagnoses, but only

one participant was an amputee and a prosthetic user. A systematic review of lower limb

prosthetic outcome measures, by Condie (111) concluded that generic function and quality of life

measures are not appropriate for lower limb amputees. Future studies are needed to more closely

evaluate the suitability of the GOAL for this LLA sub-group and compare its performance with

them to other amputee-specific outcome measures such as AMP. Amputee-specific measures

tend to include items that pertain specifically to the fit and use of a prosthetic device and may

compare performance with and without prosthetic wear. Although such measures specifically

target issues that are unique to these children, there is value in using a more generic measure

such as the GOAL, as it increases the opportunity for comparison of results across the LLA

spectrum. In addition, children with acute illness related to sarcoma were excluded from our

64

study because it was anticipated the comorbidities of their condition (e.g., fatigue) might

influence their day-to-day performance, making the suitability of the GOAL-LLA to this LLA

sub-group uncertain. In the future the suitability of the GOAL-LLA will also need to be

evaluated with these children.

Although all of the key HCPs that work with children with LLAs were represented in our study

(i.e., orthopaedic surgeons, PTs, and nurses), our sample comprised primarily orthopaedic

surgeons (81%). It is not surprising that surgeons are over-represented in this sample, since they

are the key HCP in the current model of care to inform treatment, and most often serve as

primary investigators in research initiatives involving this population. More feedback however,

from physical therapists and nurses, may be warranted. In addition, orthotists/prosthetists,

psychologists, and social workers were not represented in this study sample. Further consultation

with these professionals could possibly generate new item suggestions.

The response scale was adjusted after the second administration of the questionnaire (GOAL-

LLA 1.0). Using word descriptors as anchors for only the end values (highest and lowest), the

development team felt that, given the odd number of response options, a neutral midpoint had

been created where it did not previously exist. Specific feedback on this new scale design still

needs to be sought to ensure it is actually an improvement compared to the original version.

Asking respondents to put words to the response options that are unlabeled will reveal their

comprehension of the scale and its intervals, and their perception of midpoint neutrality.

In an effort to optimize recruitment there were two different methods used for the administration

of the GOAL. The questionnaire was completed either in clinic in the presence of an investigator

available to provide support and ensure that parents and children worked independently, or at

home. There is no way of knowing if the children and parents completed the GOAL

independently in the home environment even though we asked them to do so on their own.

However, in total, only two families (less than 9% of the sample size) completed the

questionnaire at home and mailed in their responses so the effect of inconsistent administration

was considered negligible.

The GOAL is intended to be self-administered. However, for this study the questionnaire was

administered at the point of care by the investigator to ensure that the child and parent worked

independently, and to be available to respond to (and keep detailed notes on) any questions that

65

arose. The opportunity for respondents to ask questions and seek clarification when needed

might decrease the likelihood of missing item responses. Additionally, having the investigator in

the same room might increase a child and/or parent’s sense of accountability, making them more

likely to respond to each item but might decrease the respondent’s sense of anonymity making

them more likely to respond in a way they perceive as socially desirable. Additionally, this

increased sense of accountability may have minimized the time required to complete the

questionnaire (between 12 to 19 minutes) compared to self-administration.

The guiding principle used to eliminate items (i.e., “items were considered for elimination if both

the child and parent mean item score was within 0.5 of the maximum score (e.g., 5.5 to 6.0 out of

6.0) or if both the child and parent mean importance rating was from 0 to 0.5 out of 6.0”) led to

some inconsistencies in the decision-making process in regard to which items were retained or

eliminated. The research team (JD and UN) was cautious in their elimination of items,

recognizing that each administration of the questionnaire was to a relatively small sample [n= 12

children and 11 parents (GOAL-CP), n= 5 children and 5 parents (GOAL-LLA 1.0), and n= 7

children and 6 parents (GOAL-LLA 1.1)]. Additionally, the elimination process was

complicated by the misinterpretation of importance ratings by some respondents, as previously

discussed, casting some doubt on the validity of these scores to inform item elimination in the

manner that the research team intended. To overcome these limitations, the team often opted to

retain an item until it had performed poorly in two subsequent administrations (versions) of the

questionnaire, allowing for input from a larger sample. As a result, some items that performed

poorly on one administration were ultimately retained (e.g., going up and down stairs).

The study methodology provided families the opportunity to guide the preliminary adaptations of

the questionnaire (GOAL-CP was modified to GOAL-LLA 1.0, that was then modified to

GOAL-LLA 1.1) and HCPs the opportunity to guide the final adaptations (GOAL-LLA 1.1 was

modified to GOAL-LLA 2.0). Unlike the first two adaptations of the questionnaire, the

performance of the final HCP-guided adaptation (GOAL-LLA 2.0) was not evaluated with

children and parents. However, it is reassuring that that the content between the third and final

iteration was quite stable, and any concerns will be picked up during the future formal validation

of the GOAL-LLA 2.0. The item focus was retained in 44 of 48 items from the GOAL-LLA 1.1

to GOAL-LLA 2.0, albeit with slight modifications to 12 of these items (e.g., standing in long

lineups was modified to standing for a long time (lineups/ queues, parade, concert) (Table 9).

66

Until the performance of the GOAL-LLA 2.0 is evaluated, we cannot know for certain if the

final adaptations will fully resonate with children having LLAs and their parents, and if not, what

final revisions might further improve this questionnaire for future use in this population.

8.2 Future Directions

Firstly, it would be beneficial to pilot the GOAL-LLA 2.0 on a sample of children and parents

across Canada to ensure there are no unanticipated issues considering, as noted above, that the

final questionnaire revision (GOAL-LLA 1.1 modified to GOAL-LLA 2.0) was guided primarily

by HCP feedback. This would provide an opportunity for the research team to make final

revisions to the questionnaire in preparation for formal psychometric testing.

Appreciating the time constraints and the limited support staff that HCPs might have to assist

with administration of questionnaires, our development team is committed to creating an

electronic (tablet or web-based) version of the GOAL to facilitate future clinical and research

use. This is considered a priority and should also precede formal psychometric testing so that the

electronic and paper versions of this questionnaire can be validated concurrently.

Formal psychometric testing of the GOAL-LLA is required prior to its utilization as an outcome

measure in research or clinical practice. These studies should evaluate reliability (internal

consistency and test-retest), validity (construct), and responsiveness (sensitivity to change).

Additionally, a larger sample of amputees and inclusion of children with sarcoma is required to

ensure the GOAL-LLA 2.0 is suitable for these LLA sub-groups. High internal consistency

(Cronbach’s alpha >90%) (112) within a specific domain may be suggestive of item redundancy,

indicating that further item elimination should be considered.

A confirmatory factor analysis is recommended to evaluate the development team’s decision to

group certain items together by domain based on subjective judgments. Factor analysis may

further highlight items that should be considered for elimination (e.g., items that do not load on

to any factor [i.e., domain]).

Given that the paediatric LLA population that requires treatment is small, and that the final

results of some surgical interventions (e.g., EFT, epiphyseodesis) may not be fully appreciated

for several months to years, the most practical study design would be to combine several

67

elements of psychometric testing together (i.e., reliability, validity, and responsiveness) and use a

large, international cohort, that can be studied both cross-sectionally and longitudinally.

8.3 Conclusions

This study supports the introduction of the GOAL-LLA 2.0 as a promising new outcome

measure for comprehensively evaluating the physical and psychosocial gait-related function and

priorities of children with LLAs, across all domains of the ICF, for both research and clinical

purposes. The methodology employed ensures this questionnaire is sensible for this population

and in particular carries excellent face and content validity. Following successful psychometric

testing, the GOAL-LLA has the potential to fill an identified gap in outcome measurement for

this population. Furthermore, it should fit well with family centered, team based care, providing

an opportunity for family priorities to be formally identified and inform individualized goal-

setting, intervention strategies, and outcome evaluation.

68

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Functional Health Questionnaire: Report on Reliability, Validity, and Sensitivity to Change.

Journal of Pediatric Orthopedics. 1998;18:561-71.

80. Das S, Ganesh G, S P, Mohanty R. Outcome of eight-plate hemiepiphysiodesis on genu

valgum and height correction in bilateral fibular hemimelia. Journal of Pediatric Orthopedics.

2014;23:67-72.

81. Eralp L, Kocaoglu M, Toker B, Balci H, Awad A. Comparison of fixator-assisted aniling

versus circular external fixator for bone realignment of lower extremity anular deformities in

rickets disease. Archives of Orthopaedic & Trauma Surgery. 2011;131:581-9.

82. Liu T, Zhang X, Li Z, Peng D. Management of combined bone defect and limb-length

discrepancy after tibial chronic osteomyelitis. Orthopedics. 2011;34:e363-7.

83. Marshall M, Lockwood A, Bradley C, Adams C, Joy C, Fenton M. Unpublished rating

scales: a major source of bias in randomized controlled trials of treatments for schizophrenia.

British Journal of Psychiatry. 2000;176:249-52.

84. Karpati F, Narayanan U, Weir S. Gait Outcomes Assessment List (GOAL): Further

development of a meaningful outcome measure for children with ambulatory Cerebral Palsy. 6th

Annual Bloorview Research Symposium; Holland Bloorview Kids Rehabilitation Hospital2011.

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85. Narayanan U, Weir S, Davidson B, editors. Gait Outcomes Assessment List (GOAL):

Developing a meaningful outcome measure for children with ambulatory cerebral palsy. 65th

Annual Meeting of American Academy for Cerebral Palsy and Developmental Medicine

(AACPDM); 2011; Las Vegas, Nevada.

86. Guyatt G, Bombardier C, Tugwell P. Measuring disease-specific quality life in clinical

trials. Canadian Medical Association Journal. 1986;134:889-95.

87. Ho E, Narayanan U, editors. Further development of the Gait Outcomes Assessment List

(GOAL): A patient priority-based outcome measure for ambulatory children with cerebral palsy.

Hospital for Sick Children Reasearch Day; June 26, 2013; Toronto, ON.

88. Rowe B, Oxman A. An assessment of the sensibility of a quality-of-life instrument. Am J

Emerg Med. 1993;11:374-80.

89. Feinstein A. Clinimetrics. New Haven, CT: Yale University Press; 1987.

90. O'Brien KK, Bayoumi AM, Bereket T, Swinton M, Alexander R, King K, et al.

Sensibility assessment of the HIV Disability Questionnaire. Disability and rehabilitation.

2013;35:566-77.

91. Salbach N, Jaglal S, Williams J. Reliability and validity of the Evidence-Based Practice

Confidence (EPIC) Scale. Continuing Education in the Health Professions. 2013;33:33-40.

92. Terwee CB, Mokkink LB, van Poppel M, Chinapaw M, van Mechelen W, de Vet H.

Qualitative attributes and measurement properties of physical activity questionnaires: A

checklist. Sports Med. 2010;40:525-37.

93. Lohr K, Aaronson N, Alonso J, Burman M, Patrick D, Perrin E, et al. Evaluating quality-

of-life and health status instruments: development of scientific review criteria. Clinical

Therapeutics. 1996;18:979-92.

94. Scientific Advisory Committee of the Medical Outcomes Trust. Assessing health status

and quality of life instruments: Attributes and review criteria. Quality of life research : an

international journal of quality of life aspects of treatment, care and rehabilitation. 2002;11:193-

205.

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related quality-of-life assessment in children and adolescents. Pharmoeconomics. 2006;24:1199-

220.

96. Wooley M, Bowen G, Bowen N. Cognitive pretesting and the developmental validity of

child self-report instruments: theory and application. Res Soc Work Pract. 2004;14:191-200.

97. Mulcahey M, Chafetz R, Santangelo A, Costello K, Merenda L, Calhoun C, et al.

Cognitive Testing of the Spinal Appearance Questionnaire with Typically Developing Youth and

Youth with Idiopathic Scoliosis. J Ped Ortho. 2011;31:661-7.

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98. Kelly L, Jenkinson C, Ziebland S. Measuring the effects of online health information for

patients: Item generation for an e-health impact questionnaire Patient Education and Couseling.

2013;93:433-8.

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parent proxy-reports of function and quality of life of children with below-the-elbow deficiency.

Journal of Bone & Joint Surgery. 2009;91:2852-9.

100. Narayanan U. Concerns, Desires, and Expectations of for Adolescent Idiopathic

Scoliosis: A Comparison of Patients', Parents', and Surgeons' Perspectives. Toronto: University

of Toronto; 2008.

101. Forrest C, Riley A, Vivier P. Predictors of children's healthcare use: the value of child

versus parental perspectives on healthcare needs. Medical Care. 2004;42:232-8.

102. Jobe J, Mingay D. Cognition and survey measurement: History and overview. Appl Cogn

Pyschol. 1991;5:175-92.

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R Foundation for Statistical Computing; 2014. Available from: http://www.R-project.org.

104. Young N, Bradley C, Blanchette V, Wakefield C, Barnard D, Wu J, et al. Development

of a health-related quality of life measure for boys with haemophilia: the Canadian Haemophilia

Outcomes- Kids Life Assessment Tool (CHO-KLAT). Haemophilia. 2004;10(Supplement 1):34-

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105. Turner R, Quittner A, Parasuraman B, Kallich J, Cleeland C, Group MFP-ROCM.

Patient-reported outcomes: Instrument development and selection issues. Internation Society for

Pharmoeconomics and outcomes research (ISPOR). 2007;10(Suppl 2):S86-93.

106. Cohen J. Statistical power analysis for the behavioral sciences. 2nd ed. New Jersey:

Lawrence Erlbaum; 1988.

107. Bond J, Kent G, Binney V, Saleh M. Psychological adjustment of children awaiting limb

reconstruction. Child: Care, Health, and Development. 1999;25:313-21.

108. Moseley C. Leg-Length discrepancy. In: Weinstein S, editor. Lovell and Winter's

Pediatric Orthopaedics, 6th Edition. Philadelphia, PA: Lippincott Williams & Wilkins; 2006. p.

1214-51.

109. Varni J, Setoguchi Y. Correlates of perceived physical appearance in children with

congenital/acquired limb deficiencies. Developmental and Behavioral Pediatrics. 1991;12:171-6.

110. DeVellis R. A consumer's guide to finding, evaluating, and reporting on measurement

instruments. Arthritis Care & Research. 1996;9:239-45.

111. Stucki G, Kostanjsek N, Ustun B, Cieza A. ICF-based classification and measurement of

functioning. European journal of physical and rehabilitation medicine. 2008;44:14.

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1991.

113. Matza L, Swensen A, Flood E, Secnik K, Leidy N. Assessment of health-related quality

of life in children: a review of conceptual, methodological, and regulatory issues. ISPOR.

2004;7:79-92.

114. Mokkink LB, Terwee CB, Patrick DL, Alonso J, Stratford PW, Knol DL, et al. The

COSMIN checklist for assessing the methodological quality of studies on measurement

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life research : an international journal of quality of life aspects of treatment, care and

rehabilitation. 2010;19:539-49.

77

Appendices

Appendix 1. Literature Search Strategy

Searches Results exp Leg Length Inequality/ae, cn, px, rh, su, th [Adverse Effects, Congenital, Psychology, Rehabilitation, Surgery, Therapy]

1399

exp Limb Deformities, Congenital/cl, px, rh, su, th [Classification, Psychology, Rehabilitation, Surgery, Therapy]

4982

exp Osteogenesis, Distraction/ae, cl, px, rh [Adverse Effects, Classification, Psychology, Rehabilitation]

326

exp Bone Lengthening/ae, px, rh [Adverse Effects, Psychology, Rehabilitation]

715

exp External Fixators/ae, sn [Adverse Effects, Statistics & Numerical Data]

312

exp Ilizarov Technique/ae, px, rh, sn [Adverse Effects, Psychology, Rehabilitation, Statistics & Numerical Data]

143

exp Femur/ab [Abnormalities] 1273

exp Tibia/ab [Abnormalities] 1105

exp Genu Varum/cn, rh, su, th [Congenital, Rehabilitation, Surgery, Therapy]

48

exp Genu Valgum/rh, su, th [Rehabilitation, Surgery, Therapy]

43

exp Amputation/ae, cn, px, rh, sn, su, th [Adverse Effects, Congenital, Psychology, Rehabilitation, Statistics & Numerical Data, Surgery, Therapy]

3530

exp Artificial Limbs/ae, px, rh, sn [Adverse Effects, Psychology, Rehabilitation, Statistics & Numerical Data]

465

1 or 2 or 3 or 4 or 5 or 6 or 7 or 8 or 9 or 10 or 11 or 12

12866

exp Questionnaires/cl, ec, mt, st, td, ut [Classification, Economics, Methods, Standards, Trends, Utilization]

10370

mobility.mp. 110706

function.mp. 1526202

14 or 15 or 16 1631879

13 and 17 1280

limit 18 to (yr="2000 - 2014" and "all child (0 to 18 years)")

435

78

Appendix 2. Development of the Gait Outcomes Assessment List (GOAL)

Item Generation

The GOAL’s initial bank of 60 potential items for children with CP, was generated through a

purposeful method of item generation from: 1) a literature review 2) appraisal of existing

questionnaires including the Gillette Gait Index (GGI), Gait Deviation Index (GDI), Movement

Analysis Profile (MAP), Gait Profile Score (GPS), Gross Motor Function Measure (GMFM-66),

Activity Scale for Kids (ASK), Pediatric Outcomes Data Collection Instrument (PODCI),

Gillette Functional Assessment Questionnaire (FAQ), Functional Mobility Scale (FMS), and the

Cerebral Palsy Quality of Life (CP-QOL), and 3) open-ended interviews with children having CP

(n=22, 15 boys, 7 girls) and their parents recruited from a paediatric rehabilitation hospital.

Appraisal of existing instruments, as was done with the GOAL, is considered a core component

of item generation as it serves two purposes: first, to ensure an appropriate measure does not

already exist, and two, to identify common themes or domains in similar or related measures

(86). Not all of the questionnaires appraised for item generation where CP-specific (e.g., the

PODCI is a generic measure for musculoskeletal and health function), increasing the likelihood

that items might be generalizable to the LLA population.

Inclusion of the target population during item generation provides valuable insight into

stakeholders’ perspectives, ensuring items correspond directly to their experiences and activities

(64, 113) and is considered a quality measure when reviewing the psychometric properties of an

outcome measure (94, 114). However, when a questionnaire is to be used in a population other

than the one for which it is developed, all items must be assessed for relevance with the new

population (114). As such, this thesis study includes children with LLAs and parents to provide

feedback on item content.

Item Elimination

The preliminary a pool of 60 items was subsequently reduced to 48 through the use of item

importance ratings. Children with CP (n=9) and their parents (n=10), recruited from a large

urban paediatric rehabilitation hospital completed the GOAL and rated the perceived importance

of each item using a 5-point scale (0=not a goal, 4=important goal). Items with average

79

importance ratings less than 1.5 were considered for elimination (84, 85). This thesis study

employed a similar methodology to evaluate the importance of the items in the GOAL to

children with LLAs and their parents, for the purpose of informing item elimination.

Item Consensus

GOAL content was further informed by a formal consensus method in which feedback was

obtained on the GOAL 2.0 from an international multi-disciplinary group of content-related

health care professionals (HCP), including developmental paediatricians, orthopaedic surgeons,

engineers, physical therapists, occupational therapists, kinesiologists, physiatrists, and orthotists.

An e-survey was sent to 187 HCPs (response rate 32%, n=60) whom were asked, for each item

of the questionnaire, to accept, accept with modification and state the modification, or reject and

provide rationale for rejection. Consultation with HCPs or an expert panel is a basic expectation

of measure development (86, 105) to ensure comprehensive coverage of all possible, relevant

signs/symptoms most often reported by patients and items that may not be considered/reported

by children (105).

80

Appendix 3- GOAL-CP

1. We are trying to learn more about how you walk and what is important to you and your mobility. 2. Please read the instructions on each page carefully. 3. Please answer all questions by circling the number and checking the box that fits best.

4. You may choose to add more items that are important to you at the end of the questionnaire.

For example:

A) Activities of Daily Living & Independence LEVEL of ASSISTANCE IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past 4 weeks; AND 2) Choose how much assistance you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities.

TOTA

L

MO

DER

ATE

MIN

IMA

L O

R

SUP

ERV

ISED

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Extremely Difficult /

Impossible Very

Difficult Difficult Slightly Difficult Easy Very Easy

No problem

at all

1. Getting in and out of bed

0 1 2 3 4

6 0

2 3

In the above example, getting in and out of bed was rated as very easy; required a moderate level of assistance; & improving this is a very important goal.

Your Name: _______________________________________________________________

Date of Completion (dd/mm/yy): _____________________________________________

5 1

GOAL Gait Outcomes Assessment List

Child Version

Page 1

81

A) Activities of Daily Living & Independence LEVEL of ASSISTANCE IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past 4 weeks; AND 2) Choose how much assistance you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities.

TOTA

L

MO

DER

ATE

MIN

IMA

L O

R

SUP

ERV

ISED

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Extremely Difficult /

Impossible Very

Difficult Difficult Slightly Difficult Easy

Very Easy

No problem at

all

1. Getting in and out of bed

0 1 2 3 4 5 6 0 1 2 3

2. Getting in and out of a chair or wheelchair

0 1 2 3 4 5 6 0 1 2 3

3. Standing at a sink or counter

0 1 2 3 4 5 6 0 1 2 3

4. Showering or bathing

0 1 2 3 4 5 6 0 1 2 3

5. Using the toilet

0 1 2 3 4 5 6 0 1 2 3

6. Getting dressed

0 1 2 3 4 5 6 0 1 2 3

7. Carrying an object while walking (eg. toy, doll, book, cell-phone)

0 1 2 3 4 5 6 0 1 2 3

8. Opening a door manually

0 1 2 3 4 5 6 0 1 2 3

9. Picking up an object off the floor

0 1 2 3 4 5 6 0 1 2 3

10. Getting in and out of a vehicle (eg. unmodified car, van or bus)

0 1 2 3 4 5 6 0 1 2 3

82

B) Gait Function & Mobility WALKING AID REQUIRED IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past 4 weeks; AND 2) Choose what walking aid you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities.

WH

EELC

HA

IR

WA

LKER

TWO

CA

NES

OR

CR

UTC

HES

ON

E C

AN

E /C

RU

TCH

/HA

ND

SU

PP

OR

T/ R

AIL

ING

OR

WA

LL

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

eme

ly im

po

rtan

t

During the past 4 weeks: Extremely

Difficult / Impossible

Very Difficult Difficult

Slightly Difficult Easy Very Easy

No problem at

all

11. Walking for more than 250 meters (around 2 blocks or 2 football fields)

0 1 2 3 4 5 6 0 1 2 3 4

12. Getting around at school (indoors) 0 1 2 3 4 5 6 0 1 2 3 4

13. Getting around at home

0 1 2 3 4 5 6 0 1 2 3 4

14. Walking for more than 15 minutes

0 1 2 3 4 5 6 0 1 2 3 4

15. Walking faster than usual to keep up with others

0 1 2 3 4 5 6 0 1 2 3 4

16. Stepping around or avoiding obstacles

0 1 2 3 4 5 6 0 1 2 3 4

17. Going up and down stairs

0 1 2 3 4 5 6 0 1 2 3 4

18. Going up and down slopes

0 1 2 3 4 5 6 0 1 2 3 4

19. Walking on uneven ground (rough, rocky, sandy)

0 1 2 3 4 5 6 0 1 2 3 4

20. Walking on slippery or icy surfaces

0 1 2 3 4 5 6 0 1 2 3 4

83

C) Pain / Discomfort / Fatigue INTENSITY IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how often you experienced pain or discomfort or tiredness in the past 4 weeks; AND 2) Choose how severe the pain or discomfort was; AND 3) Select how important a goal it is for you to reduce pain or discomfort or tiredness in each of the following.

SEV

ERE

MO

DER

ATE

MIL

D

NO

NE

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

im

po

rtan

t

During the past 4 weeks:

Every Day

Very Often (nearly every

day)

Fairly Often (2 to 3 times

a week)

A Few Times

(once a week)

Once or Twice

None of the Time

21. Pain or discomfort in my feet or ankles

0 1 2 3 4 5 0 1 2 3

22. Pain or discomfort in my lower legs

0 1 2 3 4 5 0 1 2 3

23. Pain or discomfort in my knees

0 1 2 3 4 5 0 1 2 3

24. Pain or discomfort in my thighs or hips

0 1 2 3 4 5 0 1 2 3

25. Pain or discomfort in my back

0 1 2 3 4 5 0 1 2 3

26. Feeling tired while walking

0 1 2 3 4 5 0 1 2 3

27. Feeling tired during any other physical activities that I usually enjoy (eg. swimming, running, horseback riding or other sport)

0 1 2 3 4 5 0 1 2 3

Other pain: _________________

0 1 2 3 4 5 0 1 2 3

84

D) Physical Activities, Games & Recreation IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past 4 weeks; AND 2) Select how important a goal it is for you to improve in each of the following activities.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

im

po

rtan

t

During the past 4 weeks:

Extremely Difficult /

Impossible Very

Difficult Difficult Slightly Difficult Easy Very Easy

No problem at all

I did not do this activity in the past 4

weeks

28. Using playground equipment (ladders, monkey bars, slides, etc.) 0 1 2 3 4 5 6

29. Running

0 1 2 3 4 5 6

30. Participating in gliding sports (eg. skating, rollerblading, skiing, skate/snowboarding)

0 1 2 3 4 5 6

31. Riding a bike or tricycle (with or without training wheels)

0 1 2 3 4 5 6

32. Swimming

0 1 2 3 4 5 6

33. Participating in sports that require running (eg. soccer, baseball, football, track)

0 1 2 3 4 5 6

34. Participating in sports that require jumping (eg. basketball, volleyball)

0 1 2 3 4 5 6

35. Participating in dance or martial arts (eg. karate, judo, taekwondo)

0 1 2 3 4 5 6

Other recreational or sporting activity: _________________

0 1 2 3 4 5 6

85

E) Gait Appearance IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how much of a problem you experienced with each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to improve in each of the following.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rta

nt

Extr

em

ely

im

po

rtan

t

During the past 4 weeks:

Extremely Difficult /

Impossible Very

Difficult Difficult Slightly Difficult Easy

Very Easy

No problem

at all 36. Walking with my feet flat on the ground

0 1 2 3 4 5 6

37. Walking taller or more upright (less crouched or bent at the knees)

0 1 2 3 4 5 6

38. Walking with my feet pointing straight ahead

0 1 2 3 4 5 6

39. Walking without dragging my feet

0 1 2 3 4 5 6

40. Walking without tripping and falling

0 1 2 3 4 5 6

41. Wearing my choice of footwear (eg. shoes, boots, sandals)

0 1 2 3 4 5 6

Other aspect of my walking: _________________

0 1 2 3 4 5 6

Page 6 Version 3.1

86

F) Use of Braces and Assistive Devices IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how you felt about using each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to reduce or eliminate your use of these devices.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks: Very

Unhappy Unhappy

Neither Happy

nor Unhappy Happy

Very Happy

42. Wearing braces or orthotics (eg. AFO)

0 1 2 3 4 I do not wear braces or

orthotics.

43. Using a walking aide (eg. walker, stick, cane, crutches)

0 1 2 3 4

I do not use any walking aides.

44. Using a wheelchair

0 1 2 3 4 I do not use a wheelchair.

87

G) Body Image & Self-Esteem IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how you felt about each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to improve in each of the following.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

im

po

rtan

t

During the past 4 weeks:

Very Unhappy Unhappy

Neither Happy nor Unhappy Happy Very Happy

45. The shape and position of my legs

0 1 2 3 4 46. The shape and position of my feet

0 1 2 3 4 47. The symmetry of my legs (in length and size)

0 1 2 3 4

48. The appearance of how I get around compared with others

0 1 2 3 4

49. The way others feel about how I get around

0 1 2 3 4 50. How I am treated by others

0 1 2 3 4

Page 7

88

Other Goals IMPORTANCE of GOAL

If there are any other goals (long or short term) that we have missed, please list them below AND Select how important a goal it is for you to improve in each.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

Other Goals:

1.

2.

3.

4.

5.

Comments & Suggestions

Thank You Page 8 Version 3.1

89

Appendix 4. Child Consent Form

Title of Research Project: Priorities of Patients with Musculoskeletal Conditions and Related Gait Disorders

Investigator(s):

Dr. Unni Narayanan Co-Principal Investigator

Division of Orthopaedic Surgery, The Hospital for Sick Children

416-813-6432

Dr. Virginia Wright Co-Principal Investigator

Holland Bloorview Kids Rehabilitation Hospital

416-424-3824

Jennifer Dermott Investigator

Division of Physiotherapy, The Hospital for Sick Children

416-813-6755 x48

MSc candidate (Supervisor: Dr. Unni Narayanan)

University of Toronto, Graduate Department of Rehabilitation Science

Dr. Simon Kelley Investigator

Division of Orthopaedic Surgery, The Hospital for Sick Children

416-813-6435

Catharine Bradley Investigator

Division of Physiotherapy, The Hospital for Sick Children

416-813-8414

Dr. Nancy Salbach Investigator

Graduate Department of Rehabilitation Science

The University of Toronto

416-946-8558

Purpose of the Research: The purpose of this study is to help us understand how your orthopaedic condition affects your

daily life. We want to know what aspects of your condition concern or bother you, if at all, and

what goals you might wish from the treatments that you have received, or might receive in the

future. This will help guide treatments (including surgery) by identifying the goals that are most

important to you, and also help us measure whether our treatments actually achieve those goals.

Description of the Research: This study involves one meeting with a researcher that may take place the same day as a regular

clinic visit. If this is not convenient, you can still participate in the study. A researcher will work

with you to find a good time to follow-up by telephone or have you return to the hospital,

whichever you prefer.

For part 1 of this study, you will be asked to complete a questionnaire called the Gait Outcomes

Assessment List (GOAL). You will be asked how important each question is to you as a goal,

90

and if there is anything important that you feel is missing. This will take about 30 minutes of

your time.

If you choose to participate in part 2 of this study, you will be interviewed by a researcher who

will ask you questions about what you did and did not like or understand about the GOAL and

how we could make it a better questionnaire. This interview will be recorded, if you agree to this

(separate consent form required). Part 2 of this study will take approximately 30 minutes to

complete.

A review of your medical chart will be conducted to confirm your exact leg condition and

medical history.

Potential Harms: This study will not help or harm you in anyway. We know of no harm that taking part in this

study could cause you, other than the inconvenience of the time you spend completing it.

Potential Discomforts or Inconvenience:

Participation in the study will require between 30-60 minutes of your time.

Potential Benefits:

To individual subjects:

There will be no direct benefit for taking part in this study. However, your participation would be

most valuable and much appreciated. We might learn something new about how your condition

concerns you that your doctor might not know. If so, we will ask you whether we can share that

information with your doctor to help you with that problem.

To society:

We hope that this study will help other children in the future who have leg conditions that change

how they walk. The results may lead to a better way to measure the impact of these conditions on

their daily lives and help us to make decisions about which treatments to choose to address their

goals, by better understanding of which treatments are most likely to achieve these goals.

Confidentiality: We will respect your privacy. No information about who you are will be given to anyone or be

published without your permission, unless the law makes us do this.

For example, the law could make us give information about you

If a child has been abused

If you have an illness that could spread to others

If you or someone else talks about suicide (killing themselves), or

If the court orders us to give them the study papers

Sick Kids Clinical Research Office Monitor or the regulator of the study may see your health

record to check on the study. By signing this consent form, you agree to let these people look at

your records. We will put a copy of the signed research consent form in your health record and

provide you with a copy for your own records.

91

The data produced from this study will be stored in a secure, locked location. Only members

of the research team (and maybe those individuals described above) will have access to the data.

Following publication of the results of this study, data will be kept for 7 years then destroyed as

required by Sick Kids policy. Published study results will not reveal your identity.

Reimbursement: If your participation in this study requires any additional visits to the hospital, we will reimburse

your family for parking costs.

In recognition of your time and effort you will receive a $20 gift card to either Chapters/Indigo or

iTunes.

Participation: It is your choice to take part in this study. You may choose to complete Part 1 or Part 1 and 2 of

this study. You can stop at any time. The care you get at Sick Kids will not be affected in any

way by whether you take part in this study.

Sponsorship:

This research is funded through a University of Toronto Fellowship.

Conflict of Interest:

I, and the other research team members have no conflict of interest to declare.

Consent :

By signing this form, I agree that:

1) You have explained this study to me. You have answered all my questions.

2) You have explained the possible harms and benefits (if any) of this study.

3) I understand that I have the right not to take part in the study and the right to stop at any

time. My decision about taking part in the study will not affect my health care at Sick Kids.

4) I am free now, and in the future, to ask questions about the study.

5) I have been told that my medical records will be kept private except as described to me.

6) I understand that no information about who I am will be given to anyone outside of this

study or be published without first asking my permission.

7) I agree, or consent, to take part in this study.

I would like to be in Part 1 of this study. _______________________

Yes No Signature/ Date

I would like to be in Part 2 of this study. _____________________

Yes No Signature/Date

Printed Name of Subject & Age

92

_

______________________________

Printed Name of person who explained consent Signature of Person who explained

consent & date

_____________________________________________ _____________________________

Printed Witness’ name (if the subject/legal guardian Witness’ signature & date

does not read English)

If you have questions about your

93

Appendix 5. Determination of Child’s Capacity to Consent

Prior to seeking consent the researcher will determine if the child is capable of providing consent.

The researcher is an experienced paediatric clinician with experience determining capacity for

consent to treatment.

Assent will be sought for incapable children. Consent for these children will be sought from their

accompanying parent/guardian.

Process

Review the information letter and study details in conversation with both the child and parent

Encourage and answer all questions and re -explain any information that is not clear.

Through this conversation determine the child’s capacity. Questions to help assess capacity may

include:

o Who can be a part of this study?

o What will you do in the study? What else? Do you want to do all these things?

o What if you start and decide you don’t want to do it anymore?

o Who will know what you say?

o Why do you think we are doing this study?

o Will I use your name when I write a report?

o Do you have to do this?

o Will taking part hurt you in any way?

o Who can tell you more if you have questions?

Note , an ‘incorrect’ answer will not necessarily be construed as incapacity but will cue the

researcher to re-explain and reassess.

94

Appendix 6- Research Ethics Board Approval

95

Appendix 7. Research Ethics Board Approval- Holland Bloorview

96

Appendix 8. University of Toronto Study Approval

97

Appendix 9. Information Sheet

If you need help, your parents can help you complete this form.

Date: ______________________

Male: ☐ Female: ☐

Age: ____________

Diagnosis: __________________

Which side? Left: ☐

Right: ☐ Both:☐

Have you previously had surgery

related to your legs? Yes: ☐

No: ☐

If yes, please describe____________________

__________________________________________

Do you currently:

Use an ankle foot orthosis (AFO)? Yes: ☐ No: ☐

Wear a shoe lift? Yes: ☐ No: ☐

Use any walking aid (i.e. cane) Yes: ☐ No: ☐

98

Appendix 10. Cognitive Interview Guide

Thank you for agreeing to complete the GOAL and to give me feedback on what you think of this

questionnaire and how we can make it better. Your feedback will help make sure the GOAL asks

questions that are meaningful and important to you and that the wording of the instructions, the

questions, and the answer scale are very clear and easy to understand.

Task 1: Questionnaire completion

Please use the black pen to complete every item of the questionnaire. As you read each question,

if there are any words or sentences that you do not completely understand, please highlight these

words or sentences with the provided highlighter. If there is anything you would like me to

explain, please ask. If there is anything you would like to share your thoughts on, for example

something you really like or don’t like, let me know, either right away as you think of it, or circle

it and we can talk about it at the end, when you are finished the questionnaire. If you have any

other ideas that you think should be included in this questionnaire, please write these on the last

page of the questionnaire.

Notes: Task 2: Instructions

Let’s look at the 4 instructions at the top of page 1.

Are these easy to understand? Notes: What does the word ‘mobility’ mean to you? Notes: Let’s look at the instructions at the top of page 2.

In your own words can you explain what is being asked of you? Notes: What does the phrase “usually” mean to you?

Task 3: Scale

Let’s take a look at the possible answers listed across the top of page 3.

There are 7 choices ranging from extremely difficult/impossible (0) to no problem at all (6).

Did you feel you were able to find your answer in the list of possible answers listed?

Do you think there are too many options, not enough options, or is it just right? (if they answer

too many or not enough, how would you change it?)

99 Notes: How would you describe the difference between’ very difficult’ and ‘extremely difficult’? Notes: Now let’s look at the importance rating scale on this page. There are 5 choices ranging from ‘not

a goal’ to ‘extremely important’.

Are there too many options, not enough options, or is it just right? (if too many or not enough,

how would they change it) Notes: Let’s look at the scale on page 5. Notice that this now includes a new column, “I did not do this

activity in the past 4 weeks”.

Do you think this is a good or bad option to include in the scale? Why? Notes: Now let’s look at page 7. This scale is asking about your feelings. You have 5 choices from

very (0) unhappy to (4) very happy.

Are there too many options, not enough options, or is it just right? (if too many or not enough,

how would you change it?) Notes: In your own words what does “neither happy nor unhappy” mean to you? Notes: Task 4: Items

Section A: Let’s take a look at page 2.

Can you read #7 aloud? (note words of

difficulty)_____________________________________________________________________________ Can you ask the same question in your own words? Notes: The question lists “toy, doll, book, cellphone” as examples. Can you think of other examples that

you might use? Notes: Look at the next question (#8).

What does the word “manually” mean to you? Notes: Take another look at all the questions in section A.

Are there any questions that you don’t think belong in this section? Notes: Are there any questions that you would get rid of completely from this questionnaire? Notes: Is there anything you would add to this section? Notes:

100

Section B: Let’s look at page 3.

Can you read #11 aloud? (note words of

difficulty)_________________________________________________________________________

Do you feel you understand a distance of 250 m? Notes: Now Compare #11 to #14.

Explain to me the difference between these questions. Notes: Read the next question (#15).

Is there a different way to say this? Notes: Which way do you prefer?

__________________________________________________________________

Take another look at all the questions in section B.

Are there any questions that you don’t think belong in this section? Notes: Are there any questions that you would get rid of completely from this questionnaire? Notes: Is there anything you would add to this section? Notes: Section C: Let’s look at page 4.

Explain to me the difference between pain and discomfort. Notes: Can you read #27 aloud? (note words of

difficulty)___________________________________________________________________________ Is there a different way to say this? Notes: Which way do you prefer?

__________________________________________________________________

This section asks about pain/discomfort in 5 different parts of your body.

Is it easy for you to think about each of these body parts separately? Notes: Would organize or ask this group of questions any other way? Notes: Take another look at all the questions in section C.

101

Are there any questions that you don’t think belong in this section? Notes: Are there any questions that you would get rid of completely from this questionnaire? Notes: Is there anything you would add to this section? Notes: Section D: Let’s look at page 5.

Can you read #30 aloud for me? (note words of

difficulty)______________________________________________________________

What does the word ‘participating’ mean to you? Notes: What does the word ‘gliding’ mean to you? Notes: Is there another way to ask this question? Notes: Look at #33.

Is there another way to ask this question? Notes: Take another look at all the questions in section D

Are there any questions that you don’t think belong in this section? Notes: Are there any questions that you would get rid of completely from this questionnaire? Notes: Is there anything you would add to this section? Notes: Section E: Let’s look at page 6.

Look at #41.

How else could you ask this question? Notes: Look at the last line at the bottom of the page.

What does the word “aspect” mean? Notes: Is there a different word that you would use? Notes: Take another look at all the questions in section E.

Are there any questions that you don’t think belong in this section?

102 Notes: Are there any questions that you would get rid of completely from this questionnaire? Notes: Is there anything you would add to this section? Notes: Section F: Let’s look at the top of page 7.

Are there any questions that you don’t think belong in this section? Notes: Are there any questions that you would get rid of completely from this questionnaire? Notes: Is there anything you would add to this section? Notes: Section G: Let’s look at the bottom of page 7.

Can you read #47 aloud for me? (note words of

difficulty)__________________________________________

What do you think “symmetry” means? Notes: Would you ask this question(#47) any differently? Notes: Can you read #48 aloud for me?

How would you ask this question in your own words? Notes: Take another look at all the questions in section G.

Are there any questions that you don’t think belong in this section? Notes: Are there any questions that you would get rid of completely? Notes: Is there anything you would add to this section? Notes: Task 5: General How do you feel about the amount of time it took you to complete the GOAL? Notes: How easy or difficult did you find the GOAL to answer? Notes:

103

There are two purposes of the GOAL. One is to measure the impact of your leg condition on

your daily life…how well do you think the GOAL does this? Notes: And two is to highlight the areas you think are most important and would like to change…how

well do you think the GOAL does this? Notes: Do you think if your leg condition was corrected your answers would look very different? Notes: Is there anything else you can think of that would make this questionnaire better? Notes:

104

Appendix 11. Cognitive Pretesting Model

From Woolley, Bowen, Bowen, 2004, informed by DeMaio & Rothgeb (1996), Jobe & Mingay (1989), and Tourangeau, Rips, &

Rasinski, (2000).

For response options:

1. “Tell me what this means to you”

2. “Is there a different way to word this?”

3. “Do you feel there are enough/not enough options?”

For items:

1. “Will you please read the question aloud?” *

2. “Is there a different way to word this?”

3. “Do you feel you can give a good answer to that question?”

4. “Why did you pick your answer?” **

Upon completion of the questionnaire:

1. “How can we make this questionnaire better?”

2. “Do you feel there are questions too much the same?”

3. “What do you think of the length of this questionnaire?”

4. “How do you feel about the response options? Clear? Enough/not enough options?”

5. “Are the instructions clear?”

6. “Is there anything else you’d like to share?”

* Make special note of any words that children may have difficulty with.

**Any time that ‘not applicable’ is chosen, ask and record the reason for this (i.e.,

developmentally inappropriate? are they not allowed?)

105

Appendix 12. Information Letter for Healthcare Professionals

Date

Dear name of health care professional

We are writing to ask for your help in a study being conducted at SickKids and Holland

Bloorview that looks at the impact of certain orthopaedic conditions on children’s everyday life.

We want to better understand the concerns and experiences of children who have an orthopaedic

condition that affects how they walk. This will help guide treatments by identifying the goals

that are most important to them, and also help us measure whether our treatments actually

achieve those goals.

For this study you will be asked to review a questionnaire called the Gait Outcomes Assessment

List (GOAL). The Gait Outcomes Assessment List (GOAL©) has been identified as a candidate

measure to evaluate the impact of lower limb anomalies on the daily life of these children and the

effectiveness of interventions in eliciting functional change. The GOAL© was initially

developed for a pediatric ambulatory cerebral palsy population (GMFCS level I-III), to evaluate

children’s and parents’ priorities for gait interventions, as no existing outcome measure was

found appropriate for this purpose. The GOAL was developed within the context of a randomized

trial evaluating the impact of gait analysis for surgical decision making on functional outcomes

following multi-level orthopaedic surgery for children with ambulatory cerebral palsy. The

instrument was developed with a view to broadening its application to any childhood condition

associated with gait differences.

Phase one of this study consists of a content evaluation in the context of pediatric lower limb

deformities, deficiencies, and/or length discrepancies. Based on feedback from children with

lower limb anomalies and their parent(s), and cognitive debrief interviews with children a new

module of the GOAL has been created specific to orthopaedic lower limb anomalies. At this

point we are requesting input from health care professionals from different disciplines

(orthopaedic surgeons, physiotherapists, orthotists, prosthetists, etc.) who have experience in the

care of these children. For each item you will be asked to ‘Accept’, ‘Accept with modification’,

or ‘Reject’. Additionally, we would like to know if there is anything important that you feel is

missing. Participation is expected to take about 30 minutes. The survey will be conducted

106

electronically via FluidSurveys. Consent to participate will be implied by completion of the

survey.

Participation in this research is voluntary but your participation would be most valuable and

much appreciated. Even if you choose to participate in this study, you can withdraw from the

study at any time. Your privacy will be respected and all information about you will remain

confidential. You will not be identified in any presentations or publications without your

permission.

You may also contact us anytime at (416) 813-6755, x48 or by e-mail at

jennifer.dermott@sickkids.ca to let us know if you have any questions or concerns.

Thank you for taking the time to read this information. This study is an important one and your

contributions are extremely valuable.

Sincerely,

Unni G. Narayanan, MBBS, Msc, FRCS(C)

Pediatric Orthopaedic Surgeon

The Hospital for Sick Children

Simon Kelley, MBChB, FRCS

Pediatric Orthopaedic Surgeon

The Hospital for Sick Children

Virginia Wright PhD

Co-Principal Investigator

Holland Bloorview Kids Rehabilitation Hospital

Phone: 416-425-6220 ext. 35xx

Jennifer Dermott BSc(Kin), BSc(PT), MSc(c)

Physiotherapist

The Hospital for Sick Children

107

Appendix 13. GOAL-LLA 1.0

1. We are trying to learn more about how you walk and what is important to you and your mobility. 2. Please read the instructions on each page carefully. 3. Please answer all questions by circling the number and checking the box that fits best. 4. You may choose to add more items that are important to you at the end of the questionnaire.

For example:

A) Activities of Daily Living & Independence LEVEL of ASSISTANCE IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it is for you to perform each of these activities; AND 2) Choose how much assistance you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities.

TOTA

L

MO

DER

ATE

MIN

IMA

L O

R

SUP

ERV

ISED

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

Extremely Difficult /

Impossible Very

Difficult Difficult Slightly Difficult Easy

Very Easy

No problem

at all

1. Getting in and out of bed

0 1 2 3 4

6 0

2 3

In the above example, getting in and out of bed was rated as very easy; required a moderate level of assistance; & improving this is a very important goal.

Your Name: _______________________________________________________________

Date of Completion (dd/mm/yy): _____________________________________________

5 1

GOAL Gait Outcomes Assessment List

Child Version

108

A) Activities of Daily Living & Independence LEVEL of ASSISTANCE IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it is for you to perform each of these activities; AND 2) Choose how much assistance you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities.

TOTA

L

MO

DER

ATE

MIN

IMA

L O

R

SUP

ERV

ISED

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

Extremely Difficult /

Impossible Very

Difficult Difficult Slightly Difficult Easy Very Easy

No problem

at all

1. Getting in and out of bed

0 1 2 3 4 5 6 0 1 2 3

2. Getting in and out of a chair (or wheelchair)

0 1 2 3 4 5 6 0 1 2 3

3. Standing at a sink or counter

0 1 2 3 4 5 6 0 1 2 3

4. Washing/bathing myself (eg. shower or tub)

0 1 2 3 4 5 6 0 1 2 3

5. Putting your pants on one leg at a time while standing

0 1 2 3 4 5 6 0 1 2 3

6. Carrying heavy objects while walking (eg. grocery bags, school books)

0 1 2 3 4 5 6 0 1 2 3

7. Balancing objects while walking (e.g. a cup of hot chocolate, a tray of food)

0 1 2 3 4 5 6 0 1 2 3

8. Opening a door

0 1 2 3 4 5 6 0 1 2 3

9. Picking up an object off the floor

0 1 2 3 4 5 6 0 1 2 3

10. Getting in and out of a vehicle (eg. car, van or bus)

0 1 2 3 4 5 6 0 1 2 3

109

B) Function & Mobility WALKING AID REQUIRED IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it is for you to perform each of these activities; AND 2) Choose what walking aid you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities.

WH

EELC

HA

IR

WA

LKER

TWO

CA

NES

OR

CR

UTC

HES

O

NE

CA

NE

/CR

UTC

H/H

AN

D

SUP

PO

RT/

RA

ILIN

G

OR

WA

LL

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

Extremely Difficult /

Impossible Very

Difficult Difficult Slightly Difficult Easy

Very Easy

No problem

at all 11. Walking for more than 250 meters (around 2 blocks or 2 football fields)

0 1 2 3 4 5 6 0 1 2 3 4

12. Getting around at school (indoors)

0 1 2 3 4 5 6 0 1 2 3 4

13. Getting around in crowded spaces (e.g. a concert or special event, the mall)

0 1 2 3 4 5 6 0 1 2 3 4

14. Standing in long lineups

0 1 2 3 4 5 6 0 1 2 3 4 15. Walking for more than 15 minutes

0 1 2 3 4 5 6 0 1 2 3 4

16. Keeping up with my friends while walking outdoors

0 1 2 3 4 5 6 0 1 2 3 4

110

17.Moving quickly if I am in a hurry (eg. to catch a bus, late for school)

0 1 2 3 4 5 6 0 1 2 3 4

18. Stepping around or avoiding obstacles

0 1 2 3 4 5 6 0 1 2 3 4

19. Going up and down stairs

0 1 2 3 4 5 6 0 1 2 3 4

20. Going up and down slopes

0 1 2 3 4 5 6 0 1 2 3 4 21. Walking on uneven ground (rough, rocky, sandy)

0 1 2 3 4 5 6 0 1 2 3 4

22. Walking on slippery surfaces (wet or icy)

0 1 2 3 4 5 6 0 1 2 3 4

111

C) Pain / Discomfort / Fatigue INTENSITY IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how often you experienced pain or discomfort or tiredness in the past 4 weeks; AND 2) Choose how severe the pain or discomfort was; AND 3) Select how important a goal it is for you to reduce pain or discomfort or tiredness in each of the following.

SEV

ERE

MO

DER

ATE

MIL

D

NO

NE

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

im

po

rtan

t

During the past 4 weeks:

Every Day

Very Often

(nearly every day)

Fairly Often (2 to 3 times a week)

A Few Times

(once a week)

Once or Twice

None of the Time

23. Pain or discomfort in my feet or ankles

0 1 2 3 4 5 0 1 2 3

24. Pain or discomfort in my knees

0 1 2 3 4 5 0 1 2 3

25. Pain or discomfort in my thighs or hips

0 1 2 3 4 5 0 1 2 3

26. Pain or discomfort in my back

0 1 2 3 4 5 0 1 2 3

27. Feeling tired while walking

0 1 2 3 4 5 0 1 2 3

28. Feeling tired during any other physical activities that I usually enjoy (eg. swimming, running, horseback riding or other sport)

0 1 2 3 4 5 0 1 2 3

Other pain:

(please specify)

0 1 2 3 4 5 0 1 2 3

112

D) Physical Activities, Games & Recreation IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it is for you to perform each of these activities; AND 2) Select how important a goal it is for you to improve in each of the following activities.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

Extremely Difficult /

Impossible

Very Difficult Difficult

Slightly Difficult Easy

Very Easy

No problem

at all

I have never tried this activity

29. Running

0 1 2 3 4 5 6

30. Participating in gliding sports (eg. skating, rollerblading, skiing, skate/snowboarding)

0 1 2 3 4 5 6

31. Riding a bicycle

0 1 2 3 4 5 6

32. Swimming

0 1 2 3 4 5 6

33. Participating in sports that require running (eg. soccer, baseball, football, track)

0 1 2 3 4 5 6

34. Participating in sports that require jumping (eg. basketball, volleyball)

0 1 2 3 4 5 6

35. Participating in dance or martial arts (eg. karate, judo, taekwondo)

0 1 2 3 4 5 6

36. Climbing (eg. ladder or stepstool) 0 1 2 3 4 5 6

Other recreational or sporting activity:

(please specify)

0 1 2 3 4 5 6

113

E) Appearance IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how much of a problem you experience with each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to improve in each of the following.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

im

po

rtan

t

During the past 4 weeks:

Extremely Difficult /

Impossible Very

Difficult Difficult Slightly Difficult Easy

Very Easy

No problem

at all 37. Walking with my feet flat on the ground

0 1 2 3 4 5 6

38. Walking taller or more upright (less crouched or bent at the knees)

0 1 2 3 4 5 6

39. Walking with my feet pointing straight ahead

0 1 2 3 4 5 6

40. Walking without dragging my feet

0 1 2 3 4 5 6

41. Walking without tripping and falling

0 1 2 3 4 5 6

42. Walking straight and level (less leaning to the side)

0 1 2 3 4 5 6

Other aspect of my walking:

(please specify)

0 1 2 3 4 5 6

114

F) Use of Braces and Assistive Devices IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how you felt about using each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to reduce or eliminate your use of these devices.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks: Very

Unhappy Unhappy

Neither Happy

nor Unhappy Happy

Very Happy

43. Wearing braces or orthotics (eg. shoelift, AFO)

0 1 2 3 4

I have not been given a lift or brace

44. Using a walking aide (eg. walker, stick, cane, crutches)

0 1 2 3 4

I have not been given a walking aid

115

G) Body Image & Self-Esteem IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how you felt about each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to improve in each of the following.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

im

po

rtan

t

During the past 4 weeks:

Very Unhappy Unhappy

Neither Happy nor Unhappy Happy Very Happy

45. The shape and position of my legs

0 1 2 3 4 ☐ ☐ ☐ ☐ ☐ 46. The shape and position of my feet

0 1 2 3 4 ☐ ☐ ☐ ☐ ☐ 47. The symmetry of my legs (in length and size)

0 1 2 3 4 ☐ ☐ ☐ ☐ ☐ 48,Wearing my choice of clothing (eg. shorts, skirts, bathing suits)

0 1 2 3 4 ☐ ☐ ☐ ☐ ☐

49.Wearing my choice of footwear (sandals, heeled boots/shoes)

0 1 2 3 4 ☐ ☐ ☐ ☐ ☐

50. The way I get around compared with others 0 1 2 3 4 ☐ ☐ ☐ ☐ ☐ 51. The way others feel about how I get around

0 1 2 3 4 ☐ ☐ ☐ ☐ ☐ 52. How I am treated by others

0 1 2 3 4 ☐ ☐ ☐ ☐ ☐

116

Other Goals IMPORTANCE of GOAL

If there are any other goals (long or short term) that we have missed, please list them below AND Select how important a goal it is for you to improve in each.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

Other Goals:

1.

2.

3.

4.

5.

Comments & Suggestions

Thank You

117

Appendix 14. GOAL-LLA 1.1

1) We are trying to learn more about you and any goals you may have for treatment of your leg condition 2) Please read the instructions on each page carefully 3) Please answer all questions by circling the number and checking the box that fits best 4) You may choose to add more items that are important to you at the end of the questionnaire

For example:

A) Activities of Daily Living & Independence LEVEL of ASSISTANCE IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities over the past 4 weeks; AND 2) Choose how much assistance you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities.

TOTA

L

MO

DER

ATE

MIN

IMA

L O

R

SUP

ERV

ISED

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

Impossible l l l l l l

No problem

at all l

3. Balancing objects while walking (e.g., a cup of hot chocolate, a tray of food)

0 1 2 3

5 6 0 1 2

In the above example, usual performance for ‘balancing objects while walking ‘ is rated 4 out of 6; requires no assistance; and improving this is a fairly important goal.

Your Name: _______________________________________________________________

Date of Completion (dd/mm/yy): _____________________________________________

GOAL-LLA Gait Outcomes Assessment List (for Children with Lower Limb Anomalies)

Child Version

4 3

118

A) Activities of Daily Living & Independence LEVEL of ASSISTANCE IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past 4 weeks; AND 2) Choose how much assistance you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities.

TOTA

L

MO

DER

ATE

MIN

IMA

L O

R

SUP

ERV

ISED

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Impossible l l l l l l

No Problem

At All l

1. Carrying heavy objects while walking (e.g., grocery bags, several school books)

0 1 2 3 4 5 6 0 1 2 3

2. Balancing objects while walking (e.g., a cup of hot chocolate, a tray of food)

0 1 2 3 4 5 6 0 1 2 3

3. Opening a door 0 1 2 3 4 5 6 0 1 2 3 4. Picking up an object off the floor

0 1 2 3 4 5 6 0 1 2 3 5. Getting in and out of a vehicle (e.g., car, van or bus)

0 1 2 3 4 5 6 0 1 2 3

119

B) Gait Function & Mobility WALKING AID REQUIRED IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past 4 weeks; AND 2) Choose how much assistance you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities.

WH

EELC

HA

IR

WA

LKER

TWO

CA

NES

OR

CR

UTC

HES

ON

E C

AN

E /C

RU

TCH

/HA

ND

SUP

PO

RT/

RA

ILIN

G O

R W

ALL

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Impossible l l l l l l

No problem

at all l

6. Walking for more than 250 meters (around 2 blocks or 2 football fields)

0 1 2 3 4 5 6 0 1 2 3 4

7. Getting around at school (indoors) 0 1 2 3 4 5 6 0 1 2 3 4

8. Getting around in crowded spaces (e.g., a concert or special event, the mall)

0 1 2 3 4 5 6 0 1 2 3 4

9. Standing in long lineups

0 1 2 3 4 5 6 0 1 2 3 4 10. Keeping up with friends

0 1 2 3 4 5 6 0 1 2 3 4 11. Moving quickly when in a hurry (e.g., to catch a bus, late for an important event)

0 1 2 3 4 5 6 0 1 2 3 4

120

B) Gait Function & Mobility- continued WALKING AID REQUIRED IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past 4 weeks; AND 2) Choose how much assistance you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities

WH

EELC

HA

IR

WA

LKER

TWO

CA

NES

OR

CR

UTC

HES

ON

E C

AN

E /C

RU

TCH

/HA

ND

SUP

PO

RT/

RA

ILIN

G O

R W

ALL

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Impossible l l l l l l

No problem

at all l

12. Stepping around or avoiding obstacles

0 1 2 3 4 5 6 0 1 2 3 4

13. Going up and down stairs 0 1 2 3 4 5 6 0 1 2 3 4

14. Going up and down slopes

0 1 2 3 4 5 6 0 1 2 3 4 15. Walking on uneven ground (rough, rocky, sandy)

0 1 2 3 4 5 6 0 1 2 3 4

16. Walking on slippery or icy surfaces

0 1 2 3 4 5 6 0 1 2 3 4

121

C) Pain / Discomfort / Fatigue INTENSITY IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how often you experienced pain or discomfort or tiredness in the past 4 weeks; AND 2) Choose how severe the pain or discomfort was; AND 3) Select how important a goal it is for you to reduce pain or discomfort or tiredness in each of the following

SEV

ERE

MO

DER

ATE

MIL

D

NO

NE

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Every Day Nearly Every

Day 2 to 3 Times

a week Once a Week Occasionally

None of the Time

17. Pain or discomfort in the feet or ankles

0 1 2 3 4 5 0 1 2 3

18. Pain or discomfort in the knees

0 1 2 3 4 5 0 1 2 3

19. Pain or discomfort in the thighs or hips

0 1 2 3 4 5 0 1 2 3

20. Pain or discomfort in the back

0 1 2 3 4 5 0 1 2 3

21. Feeling tired while walking

0 1 2 3 4 5 0 1 2 3

22. Feeling tired during any other physical activities that I usually enjoy (e.g., swimming, running, horseback riding or other)

0 1 2 3 4 5 0 1 2 3

Other pain: _________________

0 1 2 3 4 5 0 1 2 3

122

D) Physical Activities, Games & Recreation IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past year; AND 2) Select how important a goal it is for you to improve in each of the following activities

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past year:

Impossible l l l l l l

No problem at

all l

I have never tried this activity

23. Running fast

0 1 2 3 4 5 6

24. Participating in gliding sports (e.g., skating, rollerblading, skiing, skate/snowboarding)

0 1 2 3 4 5 6

25. Riding a bicycle

0 1 2 3 4 5 6

26. Swimming

0 1 2 3 4 5 6

27. Participating in sports that require running (e.g., soccer, baseball, football, track)

0 1 2 3 4 5 6

28. Participating in sports that require jumping (e.g., basketball, volleyball)

0 1 2 3 4 5 6

29. Participating in gymnastics, dance or martial arts (eg. karate, judo, taekwondo)

0 1 2 3 4 5 6

30. Climbing (e.g., ladder or stepstool) 0 1 2 3 4 5 6

Other recreational or sporting activity: _________________

0 1 2 3 4 5 6

123

E) Gait Appearance IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how much of a problem you experienced with each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to improve in each

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

im

po

rtan

t

During the past 4 weeks:

Impossible l l l l l l

No problem

at all l

31. Walking with my feet flat on the ground

0 1 2 3 4 5 6 32. Walking taller or more upright (less crouched or bent at the knees)

0 1 2 3 4 5 6 33. Walking with my feet pointing straight ahead

0 1 2 3 4 5 6 34. Walking without a limp

0 1 2 3 4 5 6 35. Walking without tripping and falling

0 1 2 3 4 5 6 Other aspect of my walking: _________________

0 1 2 3 4 5 6

124

F) Use of Braces and Assistive Devices IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how you felt about using each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to reduce or eliminate use of these devices

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Very Unhappy

l l l l

Very Happy

l

36. A shoe lift

0 1 2 3 4

☐ I have not been prescribed a

shoe lift

☐ I choose not to wear my shoe

lift

37. A brace (e.g., AFO)

0 1 2 3 4 ☐ I have not been prescribed any

type of brace

☐ I choose not to wear my brace

38. A walking aide (e.g., walker, stick, cane, crutches)

0 1 2 3 4 ☐

I do not use any walking aides

39. A wheelchair

0 1 2 3 4 ☐ I do not use a wheelchair

40. Other assistive devices (e.g., built-up bicycle pedal or _________________)

0 1 2 3 4 ☐

I do not use any other assistive

devices

125

G) Body Image & Self-Esteem IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how you felt about each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to improve in each of the following

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Very Unhappy

l l l l Very Happy

l 41. The shape and position of my legs

0 1 2 3 4 42. The shape and position of my feet

0 1 2 3 4 43. The symmetry of my legs (in length and size)

0 1 2 3 4 44. Wearing my choice of footwear (e.g., shoes, boots, sandals)

0 1 2 3 4

45. Wearing my choice of clothing (e.g., shorts, skirts, bathing suits)

0 1 2 3 4

46. The appearance of how I get around compared with others

0 1 2 3 4

47. The way others feel about how I get around

0 1 2 3 4 48. How I am treated by others

0 1 2 3 4

126

Other Goals IMPORTANCE of GOAL

If there are any other goals (long or short term) that we have missed, please list them below AND select how important a goal it is for you to improve in each.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

Other Goals:

1.

2.

3.

4.

5.

Comments & Suggestions

Thank You

127

Appendix 15. Results of the Third Administration of the GOAL (GOAL-LLA 1.1) in Phase 1 (Child

and Parent Perspectives) Children (n = 5) Parents (n=3)

Ability

Score Mean

(range)

Importance

Rating Mean

(range)

Ability

Score Mean

(range)

Importance

Rating Mean

(range)

A) (/6) (/4) (/6) (/4)

Putting your pants on one leg at a time while

standing

5.7 (4-6) 1 (0-4) 5.3 (3-6) 2.1 (0-4)

Carrying heavy objects while walking (e.g.,

grocery bags, school books)

5.4 (2-6) 1 (0-3) 4.7 (3-6) 2.3 (0-4)

Balancing objects while walking (e.g. a cup

of hot chocolate, a tray of food)

5.0 (4-6) 1.0 (0-3) 4.7 (3-6) 2.4 (0-4)

Opening a door 6.0 (6) 0.5 (0-3) 6.0 (6) 1.7 (0-4)

Picking up an object off the floor 6.0 (6) 0.5 (0-3) 5.7 (5-6) 2.0 (0-4)

Getting in and out of a vehicle (e.g., car, van

or bus)

5.9 (5-6) 0.5 (0-3) 5.4 (4-6) 2.0 (0-4)

B) (/6) (/4) (/6) (/4)

Walking for more than 250 meters (around 2

blocks or 2 football fields)

4.0 (2-6) 2.3 (0-4) 4.4 (2-6) 2.9 (0-4)

Getting around at school (indoors) 5.9 (5-6) 1.3 (0-4) 5.1 (3-6) 2.3 (0-4)

Getting around in crowded spaces (e.g. a

concert or special event, the mall)

4.9 (2-6) 1.3 (0-4) 4.6 (2-6) 2.3 (0-4)

Standing in long lineups 4.4 (2-6) 1.8 (0-4) 4.1 (2-6) 2.7 (0-4)

Keeping up with my friends 5.1 (3-6) 1.7 (0-4) 4.1 (2-6) 3.4 (2-4)

Moving quickly if I am in a hurry (e.g., to

catch a bus, late for event)

5.3 (4-6) 1.3 (0-4) 4.1 (3-6) 2.7 (0-4)

Stepping around or avoiding obstacles 5.6 (5-6) 1.2 (0-4) 5.4 (4-6) 1.8 (0-4)

Going up and down stairs 5.9 (5-6) 1.3 (0-4) 5.0 (3-6) 2.3 (0-4)

Going up and down slopes 5.3 (5-6) 1.4 (0-4) 4.7 (3-6) 2.8 (0-4)

Walking on uneven ground (rough, rocky,

sandy)

5.0 (4-6) 1.7 (0-4) 4.1 (3-5) 2.7 (0-4)

Walking on slippery surfaces (wet or icy) 4.9 (3-6) 1.7 (0-4) 3.7 (1-5) 2.7 (0-4)

C) (/5) (/4) (/5) (/4)

Pain or discomfort in my feet or ankles 3.3 (0-5) 1.2 (0-4) 3.1 (0-5) 3.0 (0-4)

Pain or discomfort in my knees 2.3 (0-5) 2.3 (0-4) 2.6 (0-5) 3.2 (0-4)

Pain or discomfort in my thighs or hips 3.4 (0-5) 1.7 (0-4) 3.7 (0-5) 1.3 (0-4)

Pain or discomfort in my back 2.9 (0-5) 2.3 (0-4) 3.7 (0-5) 2.5 (0-4)

Feeling tired while walking 1.9 (0-4) 1.9 (0-4) 2.4 (0-5) 2.8 (0-4)

Feeling tired during any other physical

activities that I usually enjoy (e.g.,

swimming, running, horseback riding or other

sport)

3.4 (0-5) 1.2 (0-4) 3.4 (0-5) 2.6 (0-4)

D) (/6) (/4) (/6) (/4)

Running fast 3.9 (1-6) 1.2 (0-4) 3.0 (0-6) 2.3 (0-3)

Participating in gliding sports (e.g., skating,

rollerblading, skiing, skate/snowboarding)

4.6 (1-6) 1.25 (0-4) 3.8 (1-5) 2.5 (2-3)

Riding a bicycle 5.7 (5-6) 0.2 (0-1) 4.7 (0-6) 2.6 (0-4)

Swimming 5.4 (2-6) 0.3 (0-2) 5.4 (3-6) 2.6 (0-4)

Participating in sports that require running

(e.g., soccer, baseball, football, track)

3.8 (1-5) 1.8 (0-4) 2.6 (1-6) 2.3 (1-4)

Participating in sports that require jumping

(e.g., basketball, volleyball)

4.7 (2-6) 1.5 (0-4) 3.3 (1-6) 2.3 (1-4)

Participating in gymnastics, dance or martial

arts (e.g., karate, judo, taekwondo)

4.0 (1-5) 2.0 (1-4) 3.3 (0-6) 1.6 (0-2)

Climbing (e.g., ladder or stepstool) 5.6 (5-6) 0.8 (0-4) 4.3 (2-6) 2.3 (0-4)

E) (/6) (/4) (/6) (/4)

Walking with my feet flat on the ground 2.1 (0-6) 3.2 (0-4) 1.9 (0-6) 2.7 (0-4)

Walking taller or more upright (less crouched

or bent at the knees)

4.3 (2-6) 1.8 (0-4) 3.7 (1-6) 3.1 (2-4)

Walking with my feet pointing straight ahead 5.1 (3-6) 1.2 (0-4) 4.7 (3-6) 2.4 (0-4)

128

Walking without a limp 5.6 (5-6) 1.0 (0-4) 4.7 (3-6) 2.4 (0-4)

Walking without tripping and falling 5.0 (1-6) 1.0 (0-4) 5.0 (2-6) 2.3 (0-4)

F) (/4) (/4) (/4) (/4)

A shoe lift 1.4 (0-4) 3.5 (3-4)

1.8 (0-4) 3.6 (3-4)

A brace (e.g., AFO) 2.0 (0-4) 4.0 (4) 3.0 (2-4) 2.0 (0-4)

A walking aide (e.g., walker, stick, cane,

crutches)

2.5 (2-3) 4.0 (4) 3.0 (2-4) 3.0 (2-4)

A wheelchair 1.0 (0-2) 4.0 (4) 4.0 (4) 0

Other assistive devices (e.g., built-up bicycle

pedal or ____________)

G) (/4) (/4) (/4) (/4)

The shape and position of my legs 3.0 (0-4) 1.3 (0-4) 2.1 (0-4) 3.0 (0-4)

The shape and position of my feet 3.3 (0-4) 0.8 (0-4) 2.3 (1-4) 2.3 (0-4)

The symmetry of my legs (in length and size) 1.7 (0-4) 2.5 (0-4) 1.1 (0-3) 3.5 (2-4)

Wearing my choice of footwear (e.g., shoes,

boots, sandals)

1.6 (0-4) 3.0 (2-4) 1.6 (0-4) 3.1 (0-4)

Wearing my choice of clothing (e.g., shorts,

skirts, bathing suits)

3.6 (1-4) 0.8 (0-4) 3.1 (2-4) 2.4 (0-4)

The appearance of how I get around

compared with others

3.1 (1-4) 1.0 (0-4) 2.0 (0-4) 3.3 (1-4)

The way others feel about how I get around 3.0 (2-4) 1.3 (0-4) 2.9 (1-4) 2.4 (0-4)

How I am treated by others 3.7 (3-4) 1.0 (0-4) 3.4 (2-4) 2.3 (0-4)

129

Appendix 16. GOAL-LLA 2.0

1) We are trying to learn more about you and any goals you may have for treatment of your leg condition 2) Please read the instructions on each page carefully 3) Please answer all questions by circling the number and checking the box that fits best 4) You may choose to add more items that are important to you at the end of the questionnaire

For example:

A) Gait Function & Mobility WALKING AID REQUIRED IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past 4 weeks; AND 2) Choose how much assistance you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities.

WH

EELC

HA

IR

WA

LKER

TWO

CA

NES

OR

CR

UTC

HES

ON

E C

AN

E /C

RU

TCH

/HA

ND

SUP

PO

RT/

RA

ILIN

G O

R W

ALL

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Impossible

l l l l l l

No problem at all

l

1. Carrying heavy objects (e.g., grocery bags, several school books) while walking

0 1 2 3 X 5 6 0 1 2 3 X X

In the above example, usual performance for ‘carrying heavy objects ‘ is rated 4 out of 6; requires no walking aid; and improving this is a fairly important goal.

Your Name: _______________________________________________________________

Date of Completion (dd/mm/yy): _____________________________________________

GOAL-LLA Gait Outcomes Assessment List (for Children with Lower Limb Anomalies)

Child Version

130

A) Gait Function & Mobility WALKING AID REQUIRED IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past 4 weeks; AND 2) Choose how much assistance you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities.

WH

EELC

HA

IR

WA

LKER

TWO

CA

NES

OR

CR

UTC

HES

ON

E C

AN

E /C

RU

TCH

/HA

ND

SUP

PO

RT/

RA

ILIN

G O

R W

ALL

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Impossible

l l l l l l

No problem at all

l

1. Carrying heavy objects (e.g., grocery bags, several school books) while walking

0 1 2 3 4 5 6 0 1 2 3 4

2. Balancing objects (e.g., a cup of hot chocolate, a tray of food) while walking

0 1 2 3 4 5 6 0 1 2 3 4

3. Walking for more than 250 meters/820 feet (around 2 blocks or 2 football fields)

0 1 2 3 4 5 6 0 1 2 3 4

4. Getting around in crowded spaces (e.g., school, a concert or the shopping mall)

0 1 2 3 4 5 6 0 1 2 3 4

5. Standing for a long time (e.g., lineups, queues, parade)

0 1 2 3 4 5 6 0 1 2 3 4

131

A) Gait Function & Mobility- continued WALKING AID REQUIRED IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past 4 weeks; AND 2) Choose how much assistance you required to help you perform these activities; AND 3) Select how important a goal it is for you to improve in each of the following activities

WH

EELC

HA

IR

WA

LKER

TWO

CA

NES

OR

CR

UTC

HES

ON

E C

AN

E /C

RU

TCH

/HA

ND

SUP

PO

RT/

RA

ILIN

G O

R W

ALL

IND

EPEN

DEN

T

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Impossible l l l l l l

No problem

at all l

6. Keeping up with friends while walking outdoors

0 1 2 3 4 5 6 0 1 2 3 4

7. Stepping around or avoiding obstacles

0 1 2 3 4 5 6 0 1 2 3 4

8. Going up and down stairs 0 1 2 3 4 5 6 0 1 2 3 4

9. Going up and down ramps

0 1 2 3 4 5 6 0 1 2 3 4 10. Walking on uneven ground (rough, rocky, sandy)

0 1 2 3 4 5 6 0 1 2 3 4

11. Walking on wet, slippery or icy surfaces

0 1 2 3 4 5 6 0 1 2 3 4

132

B) Pain / Discomfort / Fatigue INTENSITY IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how often you experienced pain or discomfort or tiredness in the past 4 weeks; AND 2) Choose how severe the pain or discomfort was; AND 3) Select how important a goal it is for you to reduce pain or discomfort or tiredness in each of the following

SEV

ERE

MO

DER

ATE

MIL

D

NO

NE

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Every Day Nearly Every

Day 2 to 3 Times

a week Once a Week Occasionally

None of the Time

12. Pain or discomfort in the feet or ankles

0 1 2 3 4 5 0 1 2 3

13. Pain or discomfort in the lower legs (shin, calf)

0 1 2 3 4 5 0 1 2 3

14. Pain or discomfort in the knees

0 1 2 3 4 5 0 1 2 3

15. Pain or discomfort in the thighs or hips

0 1 2 3 4 5 0 1 2 3

16. Pain or discomfort in the back

0 1 2 3 4 5 0 1 2 3

17. Feeling easily tired during physical activities that I enjoy (e.g., swimming, running, or other sports)

0 1 2 3 4 5 0 1 2 3

Other pain: _________________

0 1 2 3 4 5 0 1 2 3

133

C) Physical Activities, Games & Recreation IMPORTANCE of GOAL

Consider how you usually perform each of the following activities. 1) Rate how easy or difficult it was for you to perform each of these activities in the past year; AND 2) Select how important a goal it is for you to improve in each of the following activities

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past year:

Impossible l l l l l l

No problem at

all l

I have never tried this activity

18. Running fast

0 1 2 3 4 5 6

19. Participating in gliding sports (e.g., skating, rollerblading, skiing, skate/snowboarding)

0 1 2 3 4 5 6

20. Riding a bicycle

0 1 2 3 4 5 6

21. Swimming

0 1 2 3 4 5 6

22. Participating in sports that require running (e.g., soccer, baseball, football, track)

0 1 2 3 4 5 6

23. Participating in sports that require jumping (e.g., basketball, volleyball, trampoline)

0 1 2 3 4 5 6

24. Participating in sports that require balance (gymnastics, dance or martial arts)

0 1 2 3 4 5 6

25. Climbing (ladder, playground equipment, wall climbing)

0 1 2 3 4 5 6

Other recreational or sporting activity: _________________

0 1 2 3 4 5 6

134

D) Gait Appearance IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how much of a problem you experienced with each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to improve in each

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

im

po

rtan

t

During the past 4 weeks:

Impossible l l l l l l

No problem

at all l

26. Walking with my feet flat on the ground

0 1 2 3 4 5 6 27. Walking taller or more upright (less crouched or bent at the knees)

0 1 2 3 4 5 6 28. Walking with my feet pointing straight ahead

0 1 2 3 4 5 6 29. Walking without a limp

0 1 2 3 4 5 6 30. Walking without tripping and falling

0 1 2 3 4 5 6 Other aspect of my walking: _________________

0 1 2 3 4 5 6

135

E) Use of Braces and Assistive Devices IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how you felt about using each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to reduce or eliminate use of these devices

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Very Unhappy

l l l l

Very Happy

l

31. A shoe lift

0 1 2 3 4

☐ I have not been prescribed a

shoe lift

☐ I choose not to wear my shoe

lift

32. A brace (e.g., AFO)

0 1 2 3 4 ☐ I have not been prescribed any

type of brace

☐ I choose not to wear my brace

33. A prosthesis (artificial leg) 0 1 2 3 4

☐ I have not been prescribed a

prosthesis

☐ I choose not to wear my

prosthesis

34. A walking aide (e.g., walker, stick, cane, crutches)

0 1 2 3 4 ☐

I do not use any walking aides

35. A wheelchair

0 1 2 3 4 ☐ I do not use a wheelchair

36. Other assistive devices (e.g., built-up bicycle pedal or _________________)

0 1 2 3 4 ☐

I do not use any other assistive

devices

136

F) Body Image & Self-Esteem IMPORTANCE of GOAL

Consider each of the following items. 1) Rate how you felt about each of the following in the past 4 weeks; AND 2) Select how important a goal it is for you to improve in each of the following

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

During the past 4 weeks:

Very Unhappy

l l l l Very Happy

l 37. The shape and position of my legs

0 1 2 3 4 38. The shape and position of my feet

0 1 2 3 4 39. The symmetry of my legs (in length and size)

0 1 2 3 4 40. Wearing my choice of footwear (e.g., shoes, boots, sandals)

0 1 2 3 4

41. Wearing my choice of clothing (e.g., shorts, skirts, bathing suits)

0 1 2 3 4

42. The appearance of how I get around compared with others

0 1 2 3 4

43. The way others feel about how I get around

0 1 2 3 4 44. How I am treated by others

0 1 2 3 4

137

Other Goals IMPORTANCE of GOAL

If there are any other goals (long or short term) that we have missed, please list them below AND select how important a goal it is for you to improve in each.

No

t a

goal

No

t ve

ry im

po

rtan

t

Fair

ly im

po

rtan

t

Ve

ry im

po

rtan

t

Extr

em

ely

imp

ort

ant

Other Goals:

1.

2.

3.

4.

5.

Comments & Suggestions

Thank You

138