A discrete choice experiment survey was developed based on qualitative interviews. Patients with SMA (≥18 years) and caregivers (of patients <18 years) were recruited through a patient organization. Respondents made choices in each of 12 sets of hypothetical treatments. The relative importance of five treatment characteristics was compared (measured by regression coefficients [RC] of conditional logit models): (1) improvement or stabilization of motor function, (2) improvement or stabilization of pulmonary function, (3) indication for all ages or pediatric patients only, (4) route of administration (repeated intrathecal [IT] injections, one-time intravenous infusion, daily oral delivery) and (5) potential harm (mild, moderate, serious/life-threatening).

Patient ages ranged from <1 to 67 years (N=101, 65 self-reported and 36 caregiver-reported) and 64/36% were female/male (Type 1, n=21; Type 2, n=48; Type 3, n=29; other, n=3). Spinal surgery was reported in 71 patients and nusinersen or onasemnogene abeparvovec-xioi use in 69 patients.

Improvements in motor/breathing function, broad indication, oral or one-time infusion and minimal risk were preferred treatment attributes. Treatment decision should be made in clinical context and be tailored to patient needs.