Abstract Details

Title Ambulation Status, Role Participation and Caregiver Assistance among Individuals with Spinal Muscular Atrophy Type III: Results from the 2018 Cure SMA Membership Survey

Topic Child Neurology and Developmental Neurology

Presentation(s) P1 - Poster Session 1 (5:30 PM-6:30 PM)

Poster/Presentation
Number 7-061

Objective To describe ambulation status, engagement in age-appropriate roles (school, employment), and reliance on caregivers among patients with spinal muscular atrophy (SMA) type III.

Background Although individuals with SMA type III have a normal life expectancy, many patients experience loss of ambulation, highlighting the potential for significant disease burden among patients and their families. We sought to further understand disease burden by examining educational and occupational engagement among SMA type III patients and level of caregiver assistance.

Design/Methods Data came from the 2018 Cure SMA Membership Survey of affected individuals and/or their caregivers.

Results

Analyses included 138 individuals with SMA type III (type IIIa: n=48, type IIIb: n=90). Average age at survey for type IIIa was 15.1 (SD=12.4) years, and 38.1 (SD=18.1) years for type IIIb. Approximately half of the type IIIa and IIIb patients had lost their ability to walk. Most school-aged children (5-17 years, n=42) were currently in school (type IIIa: 81.5%, type IIIb: 100%). Yet only 71.4% and 49.3% of adults with type IIIa and IIIb, respectively, were in school or employed. Nearly all type IIIa patients (97.9%) and the majority with type IIIb (86.7%) reported having a caregiver. For both SMA subtypes, a family member was most frequently identified as the full-time caregiver; about one-quarter of patients (type IIIa: 29.2%, type IIIb: 18.9%) reported having a paid caregiver. Over three-quarters of both type IIIa and IIIb patients with a paid caregiver reported needing assistance for >20 hours per week.

Conclusions Results highlight the loss of ambulation many individuals with type III face, often by adolescence for type IIIa; potential impacts on education and employment into adulthood; and the high degree of reliance on family and paid caregivers. The individual, family and potential economic impacts of SMA type III may be substantial and should be studied further.

Authors/Disclosures
Lisa Belter PRESENTER	Lisa Belter has received personal compensation for serving as an employee of Cure SMA.
	No disclosure on file
Rosangel E. Cruz, MA	Ms. Cruz has nothing to disclose.
	No disclosure on file
	No disclosure on file
Sandra P. Reyna, MD	Dr. Reyna has received personal compensation for serving as an employee of Novartis Gene Therapies.
Susan A. Hall, PhD (New England Research Institute)	No disclosure on file
Angela Paradis	Angela Paradis has received personal compensation for serving as an employee of Biogen. An immediate family member of Angela Paradis has received personal compensation for serving as an employee of Biogen. Angela Paradis has received stock or an ownership interest from Biogen. An immediate family member of Angela Paradis has received stock or an ownership interest from Biogen.

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