Transitional Care for PediatricPatients with Neuromuscular
Diseases: A Health Technology Assessment
Jackie Tran, MDUniversity of Medicine and Dentistry of New
Jersey, USA
HTAi 9th Annual Meeting, BilbaoIntegrated Care for a Patient Centered System
25 June, 2012
Outline
Background Transitional Care Overview Neuromuscular Diseases Overview
Objectives Methods Results
Barriers Recommendations
Conclusions
Transitional Care OverviewBackground
Unique Adult Needs
Physical maturation Size Endocrine management Deformities
Sexual maturation Menstruation Fertility Pregnancy
Psychosocial maturation Identity Experimentation Independence Family/Friends
Intellectual maturation Education Vocation
Reality: Unmet Needs
Increase hospitalization, particularly of ages 18-21
© Pediatrics. 2011 Jul;128(1):5-13. Epub 2011 Jun 27.Adults with chronic health conditions originating in childhood: inpatient experience in children's hospitals.
Reality: Unmet Needs
Non-adherence to medications over a lifetime in renal transplant patients
Decrease clinic attendance and an average of 1.47 health provider changes in diabetes type 1 patients
Decrease mobility in spina bifida patients during transitional years
Increase long-term morbidity and mortality in cystic fibrosis patients
Neuromuscular Diseases Overview
Background
Selected Neuromuscular Diseases
of ChildhoodCongenital
myopathies dystrophic (e.g., Duchenne) myotonic congenital
spinal muscle atrophy congenital myasthenia hereditary motor
sensory neuopathy Friedreich ataxia
Acquired
Guillain-Barré syndrome
myasthenia gravis polymyositis toxic myopathy
Epidemiology
Worldwide prevalence of disabling, inherited neuromuscular diseases: 1 in 3,500
Increased survival rate to adulthoodSome Survival Statistics in the Literature
60% of males born with Duchenne between 1983 – 1987 survived past age 24
More than 33% of Charcot-Marie-Tooth Type 2 patients live past the third decade of life
A patient with Type 2 SMA have survival rate of 68.5% at age 25
Unique Characteristics
Low recognition Apparent functional deficits Infamous progressive/regressive
courses Media portrayal Multi-organ systems involvement Large team investment
Equally Poor Transitional Care
A glaring 60% of 850 people living with muscle disease rate transition from child to adult services as “poor” or
“very poor”
Muscular Dystrophy Campaign 2008 survey
Finding Transitional Solutions
Objectives & Methods
Objectives
Identify systematic and unique barriers to a successful transition of care for adolescents with neuromuscular diseases (NMDs).
Recommend comprehensive strategies to improve the experience for patients, family members, and health providers alike.
Methods
Systematic literature review of publications between January 2000 and December 2011
Informal survey of local clinicians’ opinions
Only the beginning…Results
General Observations
Notable publication increase on transitional care over the past 5 years
Emphasis on the following diagnoses: diabetes, cancer, renal transplant, inflammatory bowel
dz cerebral palsy and spina bifida
Most prolific countries: USA, Canada and UK Honorable mentions: Australia, France, Japan,
Switzerland
All Transitional Care Publications
NMD-Relevant Publications
Number of
Publications
60+ 14
Population description
• generic: “chronic illnesses” & “special health care needs” & “congenital disabilities” & “developmental
disabilities”
• multiple diagnoses-specific publications
• inter-mix with spina bifida & cerebral palsy &
“chronic neurological disorders”
• Duchenne dystrophy
All Transitional Care Publications
NMD-Relevant Publications
Publication
Content
• highlight need for transitional care• clinician & patient survey/commentaries
• anecdote / case reports
• single-institution experience
• general critiques of current transition
processes & models
• review of medical topics on transitional ages
Outcome measure
s
• subjective reports of wellness & satisfaction
• individual programs: admissions & biomarkers
(retrospective & limited to 1 year after transition)
• health expenditure at transitional age as a secondary outcome
Models of Transitional Care
Diagnosis-focused (most common)
pediatric and adult provider collaboration
Medical home-based (least common)
primary care practitioner as coordinator
Adolescent-focused physical, physiological, social issues focused
Transition-based outreach representatives from one facility
General Barriers
Afterthought process Provider availability (absolute & time)
Coordinator responsibility Decision-making capacity Advisors resourcefulness Insurance coverage Transportation / Architectural Medical records Ventilator, dialysis
Emotional Barriers
Patient & Family neglect
abandonment
Adult team burden
unfamiliarity
Peds team attachment
over-confidence
FearAnxietyDistrust
Uncertainty
Unique Barriers &
Opportunities
Assistive technology Malpractice insurance
Insurance / resources for the very ill Association with spina bifida and/or cerebral
palsy
Even more limited provider availability Institutionalized patients Aging care providers Uncertain future
Barriers
Opportunities
Recommendations
Early notification Individualized plan
flexible but definitive date of transition assess cognitive/emotional and functional
status gradual promote of independence
Comprehensive approach patient’s and family members’ input interdisciplinary team
Recommendations
Coordinator designation Patient education
self care and disease process healthcare system navigation
Long-term planning vocation and education living situation
Resources availability
Advanced Topics
Program sustainability Provider training Transition to palliative care Socioeconomic status effects
Conclusions
All children with neuromuscular diseases need a transition plan before entering adulthood
Successful transition requires coordination of services across all providers
Especially for individuals affected by NMDs, the transition plan should incorporate patient’s and family’s long-term goals
Primary References
Abbott, D. Transition to adulthood for young men with Duchenne Muscular Dystrophy. International Journal of Integrated Care, 31 December 2009 - ISSN 1568-415
Centers for Disease Control and Prevention (CDC). Prevalence of Duchenne/Becker muscular dystrophy among males aged 5-24 years - four states, 2007. MMWR Morb Mortal Wkly Rep. 2009 Oct 16;58(40):1119-22.
Strehle EM. Long-term management of children with neuromuscular disorders. J Pediatr (Rio J). 2009 Sep-Oct;85(5):379-84. Tiffreau V, et al. Ann Readapt Med Phys. 2006 Dec;49(9):652-8. Epub 2006 Jun 27. [Transition in health care from youth to
adulthood for disabled people]. Ouyang L, et al. Health care utilization and expenditures for childrenand young adults with muscular dystrophy in a privately insured
population. J Child Neurol. 2008 Aug;23(8):883-8. Epub 2008 Apr 10. Manzur AY, Muntoni F. Diagnosis and new treatments in musculardystrophies. J Neurol Neurosurg Psychiatry. 2009 Jul;80(7):706-
14. Hill ME, Phillips MF. Service provision for adults with long-term disability: a review of services for adults with chronic
neuromuscular conditions in the United Kingdom. Neuromuscul Disord. 2006 Feb;16(2):107-12. Epub 2006 Jan 19. Nomura Y. [Care continuity for patients with myasthenia gravis during transition from childhood to adulthood]. Nihon Rinsho. 2010
Jan;68(1):39-44. Yoshioka M, et al. [Care continuity for patients with myopathy during transition of childhood to adulthood]. Nihon Rinsho. 2010
Jan;68(1):53-6. Minicozzi A. Transition to adult care: another view. Pediatr Nurs. 2000 Jul-Aug;26(4):411-2. Katz JD, et al. Parents' perception of self-advocacy of children with myositis: an anonymous online survey. Pediatr Rheumatol Online
J. 2011 Jun 7;9(1):10. Goodman DM, et al. Adults with chronic health conditions originating in childhood: inpatient experience in children's hospitals.
Pediatrics. 2011 Jul;128(1):5-13. Epub 2011 Jun 27. Rearick E. Enhancing success in transition service coordinators: use of transformational leadership. Prof Case Manag. 2007 Sep-
Oct;12(5):283-7. Parker AE, et al. Analysis of an adult Duchenne muscular dystrophy population. QJM. 2005 Oct;98(10):729-36. Epub 2005 Aug 31.
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