OBJECTIVES: Spinal muscular atrophy type 1 (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. We sought to describe sociodemographic and time costs for families and caregivers of patients with SMA1. Here, we present initial data reflecting caregiver burden in Argentina.

METHODS: An online survey was disseminated by a patient advocacy group, Families of SMA, Argentina (FAME), from February to May 2022. Eligible respondents were voluntary non–health care professional adult caregivers.

RESULTS: Twenty-two caregivers (mean age, 36.6 years) managing 23 patients with SMA1 responded. In 14/23 cases (60.9%), the patient’s mother was the primary or co-primary caregiver. Mean patient care time was 62.2 hours/week. Of the 23 patients, six (26.1%) had a caregiver who stopped working to provide care; 10 (43.5%) had a caregiver who reduced their working hours. Caregivers reported reducing their work time by an average of 18.7 hours/week. Impact to income was reported by caregivers representing 15 patients (65.2%; mean net monthly income reduction, 48.3%). Within the last 6 months, 13 patients (76.5%) had a caregiver who took days off from work for caregiving (mean, 12.1 days). Caregivers reported daily activities, physical therapy, and support in bathing as their most time-consuming activities.

CONCLUSIONS: Caregivers of patients with SMA1 in Argentina reported a substantial burden on time, employment status, and income. The high degree of care required and financial pressure created can have a substantial impact on caregiver health-related quality of life. Lost productivity associated with SMA1 imposes high direct and indirect societal burdens. Early SMA identification and early access to disease-modifying treatments may reduce these burdens and associated costs. Work is ongoing to describe caregiver burden globally.