PICKING THE RIGHT

PROMS FOR YOUR

RESEARCH

HOW TO AVOID SOME

COMMON PITFALLS

Professor Penny SchofieldSwinburne University of Technology

Acknowledgements: Professor Madeleine King

TOO MANY QUESTIONNAIRES

H E L P !

T. LUCKETT & M.T. KING, EUROPEAN JOURNAL OF CANCER 2010; 46: 3149-3157.

SIX GUIDING PRINCIPLES

1. Consider PROMs early in the design process

2. Choose a primary PROM that is as close to

illness/treatment

3. Identify candidate PROMs primarily on the grounds of

scaling and content

4. Appraise reliability, validity, responsive & ‘tra ck records’

of candidate PROMs in similar studies

5. Look ahead to practical concerns

6. Take a minimalist approach to ad hoc items

MRC FRAMEWORK FOR DEVELOPMENT &

EVALUATION OF COMPLEX INTERVENTIONS: 2008

Non-linear, interacting, flexible process

PENTAGON:PEER & NURSE SUPPORT TRIAL TO ASSIST

WOMEN IN GYNAECOLOGICAL ONCOLOGY:

A NATIONAL PHASE III TRIAL

PRINCIPLE INVESTIGATOR: PROFESSOR PENELOPE SCHOFIELD

TRIAL FUNDED BY CANCER AUSTRALIA/BEYONDBLUE AND NHMRC

INTERVENTION GOALS

Radiotherapy for gynaecological cancer can have distressing side effects, and may impact on psychosocial functioning and intimate relationships.

Goals:

• Detection of needs

• Tailored, evidence-based information

• MDT referrals

• Adherence to self-care plans (esp dilator use)

• Peer support

AIM & HYPOTHESES

Compared to usual care, intervention patients will report:

� Lower psychological distress (primary)

� Lower symptom distress,

� Better preparation for treatment

� Lower needs (informational and psychological),

� Higher quality of life

� Less psychosexual dysfunction & vaginal atrophy/narrowing

To test the effectiveness of a nurse-led psychosocialintervention with telephone-based peer support to improve outcomes for women receiving radiotherapy with curative intent for gynaecological cancer.

DESIGN

Design

Multi-site RCT with follow ups at immediately prio r to first treatment, 4 weeks, 6 months and 12 months post-tre atment

Randomisation

Post- baseline measures, patients will be randomised 1:1 to intervention or usual care

Peer call 4

(4 wks after end-of-treatment)

Nurse session 1

(Pre-treatment)

Peer call 1

(~1wk after nurse session 1)

Nurse contact with

Peer – concerns &

self-care

Referrals/info

Nurse session 2

(mid-treatment - wk 3)

Peer call 2

(~1wk after nurse session 2)

Nurse contact with

Peer – concerns &

self-care

Referrals/info

Nurse session 3

(end-of-treatment: wk6 EBRT,

or end of BCY.)

Peer call 3

(~1wk after nurse session 3)

Nurse contact with

Peer – concerns &

self-care

Referrals/info

Nurse contact with

Peer – concerns &

self-care

Referrals/info

Nurse:

•Treatment orientation

•side-effects

•self-care plan

•coaching (esp. dilator

use)

• psychosexual

rehabilitation

•MDT care co-

ordination

•Survivorship care plan

to pt & GP

One & half days of

training & ongoing

supervision

Manual includes:

•evidence based

recommendations,

•need assessment tool

•self-care brochures.

Peer:

•Empathy,

•share experiences

•encourage adherence

to self-care plan.

•Appropriate link with

the nurse, &

•provided with side-

effects management

plan.

Two days of training &

ongoing supervision

Manual includes:

•detailed guide for

phone conversations

•specific topics to cover

and •effective communication

techniques

The Intervention

Nurse session 4 (telephone)

(2wks after end-of-treatment)

ENDPOINTS

CONCEPTS VERSUS MEASURES

constructs

phenomena

domains

questionnaires

instruments

scales

What are the specific outcome constructs that will be influenced by the intervention?

What is the population of interest?

What is the time frame of interest?

Fairclough D. (2010) Design and Analysis of Quality of Life Studies in

Clinical Trials, 2nd Edition. New York: Chapman & Hall.

CHOOSING THE RIGHT

OUTCOME CONSTRUCT

HYPOTHESES

Compared to usual care, intervention patients will report:

� Lower psychological distress (primary)

� Lower symptom distress,

� Better preparation for treatment

� Lower needs (informational and psychological),

�Higher quality of life

� Less psychosexual dysfunction & vaginal atrophy/narrowing

OUTCOME CONSTRUCTS

Time point Questionnaire Constructs

Radiotherapy planning CT date. BaselineAnxiety and depression, Quality of life, psychological & informational needs; psycho-sexual and vaginal health

Immediately prior to 1st radiotherapy treatment. Follow-up 1 Anxiety and depression, Preparation for

treatment

4 weeks post-radiotherapy (post-intervention). Follow-up 2

Anxiety and depression, Quality of life, psychological & informational needs; Preparation for treatment, adherence to dilators

6 months post-radiotherapy. Follow-up 3

Anxiety and depression, Quality of life, psychological & informational needs; psycho-sexual and vaginal health; adherence to dilators

12 months post-radiotherapy. Follow-up 4

Anxiety and depression, Quality of life, psychological & informational needs; psycho-sexual and vaginal health; adherence to dilators

THE RIGHT QUESTIONNAIRE IS

THE ONE THAT:

Covers aspects of patient outcomes that are expecte d to affected by the intervention

• Consider what will be the treatment differential between the two arms

Has demonstrated validity, reliability and responsi veness to change

• Preferably in population(s) same/similar to your target population

Is not too burdensome

• Patients with low literacy; from CALD backgrounds or who are unwell may struggle

SCALING OF QLQ-C30 AND FACT-G

REVIEW ITEM AND SCALE

CONTENT

• Review the content of the questionnaires– What issues are covered by the items (questions)? – How items are combined into multi-item scales?

• Find the best match to your planned endpoints

SOCIAL FUNCTION VS WELLBEING

FACT-GEORTC QLQ-C30

PILOT TESTING

�Participant recruitment (n= 6 recruited)

�Semi-structured phone interviews throughout pre-

test for feedback on:

- Acceptability, burden, relevance & perceived

usefulness of the program

QUALITATIVE FEEDBACK

Reactions were very encouraging:

“very helpful…nothing negative, only positive things can come out of it [the program]!”

“if a person is giving of their time, and they’re prepared to listen, you know, listen to you, then how can you improve on that?”

“I don’t think you can improve on wonderful!”

Although….

Questionnaires sometimes disliked/not understood especially sexual questionnaire

50% patients preferred to have session 1 from home

Telephone sessions successful, but harder for the nurse.

OUTCOME MEASURES

HADS: Hospital Anxiety and Depression Scale; FACT-G: Functional Assessment for Cancer Therapy – General; SCNS-SF: Supportive Care Needs Survey-short form; MSAS-SF: Memorial Symptom Assessment Scale Short Form; SVQ: Sexual function-Vaginal changes Questionnaire; AQ: Adherence Questionnaire; CaTS: Cancer Treatment Survey

Time point Questionnaire Measures

Around radiotherapy planning CT date. Baseline Demographic variables, HADS, FACT-G, SCNS-SF,

MSAS-SF, SVQ.

Immediately prior to 1st radiotherapy treatment. Follow-up 1 HADS, CaTS -before treatment.

4 weeks post-radiotherapy (post-intervention). Follow-up 2 HADS, FACT-G, SCNS-SF, MSAS-SF, AQ, CaTS –

after treatment.

6 months post-radiotherapy. Follow-up 3 HADS, FACT-G, SCNS-SF, AQ, SVQ.

12 months post-radiotherapy. Follow-up 4 HADS, FACT-G, SCNS-SF, AQ, SVQ.