Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.
J Neuromuscul Dis
; 11(2): 425-442, 2024.
Article
in En
| MEDLINE
| ID: mdl-38250783
ABSTRACT
Background:
Long-term, real-world effectiveness and safety data of disease-modifying treatments for spinal muscular atrophy (SMA) are important for assessing outcomes and providing information for a larger number and broader range of SMA patients than included in clinical trials.Objective:
We sought to describe patients with SMA treated with onasemnogene abeparvovec monotherapy in the real-world setting.Methods:
RESTORE is a prospective, multicenter, multinational, observational registry that captures data from a variety of sources.Results:
Recruitment started in September 2018. As of May 23, 2022, data were available for 168 patients treated with onasemnogene abeparvovec monotherapy. Median (IQR) age at initial SMA diagnosis was 1 (0-6) month and at onasemnogene abeparvovec infusion was 3 (1-10) months. Eighty patients (47.6%) had two and 70 (41.7%) had three copies of SMN2, and 98 (58.3%) were identified by newborn screening. Infants identified by newborn screening had a lower age at final assessment (mean age 11.5 months) and greater mean final (SD) CHOP INTEND score (57.0 [10.0] points) compared with clinically diagnosed patients (23.1 months; 52.1 [8.0] points). All patients maintained/achieved motor milestones. 48.5% (nâ=â81/167) experienced at least one treatment-emergent adverse event (AE), and 31/167 patients (18.6%) experienced at least one serious AE, of which 8/31 were considered treatment-related.Conclusion:
These real-world outcomes support findings from the interventional trial program and demonstrate effectiveness of onasemnogene abeparvovec over a large patient population, which was consistent with initial clinical data and published 5-year follow-up data. Observed AEs were consistent with the established safety profile of onasemnogene abeparvovec.Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Biological Products
/
Recombinant Fusion Proteins
/
Muscular Atrophy, Spinal
/
Spinal Muscular Atrophies of Childhood
Type of study:
Diagnostic_studies
/
Prognostic_studies
Limits:
Humans
/
Infant
/
Newborn
Language:
En
Journal:
J Neuromuscul Dis
Year:
2024
Document type:
Article
Country of publication: