Outcomes of Onasemnogene Abeparvovec in Pediatric Patients with Spinal Muscular Atrophy (SMA): Findings from the Cure SMA 2020 Community Update Survey


Clinical Management

Poster Number: 20


Walter Toro, Novartis Gene Therapies, Inc., Nicole LaMarca, DNP, MSN, CPNP, PMHS, Novartis Gene Therapies, Lisa Belter, MPH, Cure SMA, Anish Patel, Novartis Gene Therapies, Min Yang, PhD, Analysis Group, Inc., Mihaela Georgieva, PhD, Analysis Group, Inc., Christopher Carley, Analysis Group, Inc., Annika Anderson, MPH, Analysis Group, Inc., Omar Dabbous, Novartis Gene Therapies

Objective: We sought to describe outcomes for SMA patients treated with onasemnogene abeparvovec (OA) from the Cure SMA 2020 Community Update Survey.
Background: Limited real-world evidence exists on outcomes for patients with SMA after treatment with OA, particularly in those treated at ?6 months of age or with four SMN2 gene copies.
Methods: Patients treated with OA at 6–23 months of age were matched 1:1 to non-OA-treated patients by age at time of survey. Patient characteristics, developmental milestones, healthcare resource utilization (HCRU), and caregiver proxy-reported EQ-5D-3L domains were summarized descriptively.
Results: We included 53 OA-treated and 53 matched non–OA-treated patients. Mean age (±SD) at time of survey was 22.9±12.7 and 22.8±13.2 months, and 62.3% and 49.1% of patients were female, respectively. SMA type was type 1 (60.3%; 73.6%), type 2 (34.0%; 17.0%), type 3 (5.7%; 0.0%), and other/unknown (0.0%; 9.4%). SMN2 gene copy number was 1 (0.0%; 7.5%), 2 (56.6%; 60.4%), 3 (30.2%; 20.8%), 4 (3.8%; 1.9%), and unknown (9.4%; 9.4%). Compared with non–OA-treated patients, more OA-treated patients achieved each milestone at time of survey, including head control (90.6%; 71.4%), rolling over (64.2%; 33.3%), sitting with support (76.5%; 45.0%), standing with assistance, (41.2%; 22.5%), walking with assistance (21.3%; 12.1%), standing alone (14.9%; 12.1%), and walking alone (12.1%; 10.0%). OA-treated patients had lower rates of hospitalization over the past 12 months (32.1%; 35.8%), lower use of tracheostomy with ventilator (5.7%; 15.1%), and were less likely to use oxygen or breathing machines for >16 hours/day (5.7%; 29.7%), at time of survey. Caregiver-proxy EQ-5D-3L reported extreme problems with mobility (45.3%; 50.9%), self-care (69.8%; 67.9%), usual activities (34.0%; 39.6%), and problems with pain/discomfort (9.4%; 13.2%) and anxiety/depression (0.0%; 3.8%).
Conclusions: Patients treated with OA at 6–23 months of age consistently demonstrated greater motor milestone achievement and lower HCRU and ventilator use compared with non-OA-treated patients.