OBJECTIVES: Nusinersen has demonstrated efficacy and effectiveness in patients with spinal muscular atrophy (SMA). More research is needed to understand the real-world benefits of nusinersen treatment. The objective of this study was to describe common SMA-related complications over time in patients treated with nusinersen using the PINC AI™ Healthcare Database.

METHODS: Patients who likely received their first nusinersen dose (index admission) between 01/01/2017 and 03/31/2021 were included. Patients were followed from index date to their last discharge date up to 09/30/2021. Nine SMA-related complications were assessed for patients with ≥ 24-month follow-up over three time periods: index-to-6-months, 6-to-12-months, and 12-to-24 months (normalized to 6-month values). The complications, defined using diagnosis and procedure codes, were pneumonia, ventilation support including tracheostomy, gastrointestinal complications including gastrostomy tube, musculoskeletal problems, support/therapy, orthopedic surgery, and other respiratory illnesses. Unadjusted descriptive analyses were conducted, and results were stratified by age: pediatric (0-17 years) and adult (18+ years).

RESULTS: The study included 56 patients with ≥ 24-month follow-up, of which 27 were pediatric and 29 were adult. The average age was 22 years old (SD = 18), and 50% were male. Patients received median (Q1 - Q3) 10 (7 - 11) doses during follow-up. The frequency with which patients had each complication decreased over time after start of nusinersen treatment for both age cohorts. Among pediatric patients, 48% used ventilation support (index-to-6-months), then 41% (6-to-12-months) and 19% (12-to-24 months). Gastrostomy tube use decreased from 70% (index-to-6-months) to 52% (6-to-12-months) and to 26% (12-to-24 months) in pediatric patients.

CONCLUSIONS: This study documented that SMA complications decreased over time with nusinersen treatment, suggesting potential improvement of healthcare resource utilization and additional clinical and economic benefits associated with treatment.