Abstract Details

Title Health Care Resource Utilization in Spinal Muscular Atrophy Type 1 Patients in Japan: Results of a Global Caregiver Survey

Topic Child Neurology and Developmental Neurology

Presentation(s) P16 - Poster Session 16 (8:00 AM-9:00 AM)

Poster/Presentation
Number 6-004

Objective We sought to describe health care resource utilization (HCRU), including hospitalizations, health care professional (HCP) visits, and device utilization, for children with SMA type 1 (SMA1) in Japan.

Background

SMA1, a rare, genetic neuromuscular disease that causes loss of both voluntary motor and bulbar functions, is usually fatal by 2 years of age if untreated. HCRU data on hospitalizations and ongoing standard multidisciplinary care for SMA1 are sparse.

Design/Methods An online survey was disseminated by the principal investigator and a patient advocacy group (Japan Network for Spinal Muscular Atrophy) from 10/2020–03/2021. Eligible respondents were voluntary non-HCP adult caregivers of patients with SMA type 1, 2, or 3 in Japan. Respondents reported HCRU over the past 6 months.

Results As of 03/05/2021, 48 caregivers (mean age, 38.7 years) managing 50 SMA1 patients responded. During their lifetimes, 28 patients (56%) underwent gastrostomy and 28 (56%) underwent tracheostomy. Median durations of hospital stays were 17 days for gastrostomy and 35 days for tracheostomy. Within the past 6 months, 37 patients (74%) spent at least one night in the hospital (average number of stays, 2.43; average duration of stay, 5.89 days). Forty-eight patients (96%) visited a pediatrician within the past 6 months, the majority (62.9%) in a hospital setting, with 59.1% reporting monthly visits. Other frequently reported HCP visits were to physiotherapists (82% of cases), non-specialist nurses (76%), and occupational therapists (58%). Frequently used devices or equipment included suction machine (80% of cases), cough assist machine (60%), breathing machine (54%), wheelchair (66%), sitting retainer (66%), and gastric feeding tube (64%).

Conclusions SMA1 in Japan is associated with substantial HCRU, highlighting the need for ongoing multidisciplinary care in these patients. Early diagnosis, facilitated by newborn screening and timely intervention with disease-modifying treatment, may reduce these burdens and associated costs.

Authors/Disclosures
PRESENTER	No disclosure on file
	No disclosure on file
	No disclosure on file
Omar Dabbous	Omar Dabbous has received personal compensation for serving as an employee of Novartis Gene Therapies. Omar Dabbous has received stock or an ownership interest from Novartis Gene Therapies.
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
Kayoko Saito	No disclosure on file

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