OBJECTIVES: Spinal muscular atrophy type 2 (SMA2) is an intermediate form of spinal muscular atrophy, characterized by progressive muscle weakness and atrophy. Evidence on the impact of disease management and support on caregivers of patients with SMA2 is limited, and therefore we sought to describe health care resource use, time costs, and out-of-pocket expenditures for the families/caregivers of these patients in Argentina.

METHODS: An online survey was disseminated through patient advocacy group FAME from February 2022 through May 2022. Eligible respondents were voluntary non–health care provider (HCP) caregivers.

RESULTS: Forty-nine caregivers (mean age, 43.6 years) managing 50 total patients with SMA2 participated in the survey. In 30/50 cases (60%), the patient’s mother was the primary caregiver. Mean patient care time was 79.7 hours/week. Of the 50 cases, 8 (16%) had a caregiver who stopped working to provide care and 17 (34%) had a caregiver who reduced their working hours (mean reduction, 13.8 hours/week). For caregivers experiencing income changes, the average income reduction per month was 42.2%. Twenty-five patients had caregivers take days off from work for caregiving in the last 6 months (mean, 22 days). Caregivers experienced out-of-pocket expenditures, including home adaptations (mean, 356,198.50 Argentine pesos). In addition, most caregivers consulted at least one HCP for their patient(s) and 11 patients (22.0%) had at least one overnight hospitalization, excluding SMA-related surgeries, in the last 6 months.

CONCLUSIONS: Caregivers of patients with SMA2 in Argentina reported a substantial impact on their time, employment status, and out-of-pocket costs, affecting their own well-being. Early identification and treatment of SMA2 may reduce associated costs and resulting impact on caregivers and patients.