Abstract Details

Title The Burden of Spinal Muscular Atrophy Type 1 (SMA1) on Caregivers in the United States (US): First Results of a Global Survey

Topic Child Neurology and Developmental Neurology

Presentation(s) P17 - Poster Session 17 (11:45 AM-12:45 PM)

Poster/Presentation
Number 6-004

Objective We sought to describe out-of-pocket expenditures and time costs, as well as health care resource utilization (HCRU), for the families/caregivers of patients with SMA1. Here, we present initial data reporting caregiver burden in the US.

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. Data on caregiver burden associated with SMA1 are sparse.

Design/Methods An online survey was disseminated by a patient advocacy group (Cure SMA) in the US in 07/2020. Eligible respondents were voluntary adult caregivers (non-health care professionals).

Results Interim analysis represents data as of 02/2021. Thirty caregivers (mean age, 37.13 years) who cared for 31 patients with SMA1 responded. In 25/31 cases (80.65%), the patient’s mother was the primary or co-primary caregiver. Mean patient care time was 85.13 hours/week. Of the 31 patients, 15 (48.39%) had a caregiver who stopped working to provide care; 10 (32.26%) had a caregiver who reduced their working hours (mean reduction, 17.73 hours/week); one (3.23%) had a caregiver who changed jobs. Impact to income was reported by 22 caregivers (70.97%; median net income reduction, $2650/month). Mean out-of-pocket costs over the last 6 months toward home renovations, at-home health care, and travel for medical appointments were $8600.03, $677.42, and $700.97, respectively. Within the last 6 months, four patients (12.9%) had ≥1 overnight hospitalizations (the majority for respiratory infection/breathing difficulties).

Conclusions Caregivers of patients with SMA1 in the US reported a substantial burden on their time, employment status, income, and out-of-pocket costs. Lost productivity and HCRU associated with SMA1 imposed greater direct and indirect societal burdens. Early SMA identification via newborn screening and early access to disease-modifying treatments may reduce these burdens and associated costs. Additional work to describe caregiver burden globally is ongoing.

Authors/Disclosures
PRESENTER	No disclosure on file
	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.

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