Abstract Details

Title Efficacy and Safety of Onasemnogene abeparvovec in Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis

Topic Child Neurology and Developmental Neurology

Presentation(s) P1 - Poster Session 1 (9:00 AM-5:00 PM)

Poster/Presentation
Number 137

Objective To evaluate the efficacy and safety of Onasemnogene abeparvovec (OA) in patients with spinal muscular atrophy (SMA).

Background SMA is a rare disease considered the leading genetic cause of infant death due to variable progressive proximally patterns of muscle atrophy and weakness.

Design/Methods We performed a systematic search and selected studies with SMA patients treated with OA. A meta-analysis of proportions was carried out to assess outcomes for efficacy and safety, and subgroup analyses were based on the number of copies of the SMN2 gene and on previous treatment with Nusinersen. We used ROBINS-I tool for assessing the risk of bias (ROB) and assessed the certainty of evidence based on the GRADE approach.

Results Eight studies with 144 participants were included for quantitative synthesis. Four studies were of moderate ROB and the remaining studies were at low ROB. The overall rate of patients with SMA who achieved an independent sitting for ≥30, ≥10 seconds and event-free survival rate at 14 months were 77%, 55% and 93%, respectively. Patients with any treatment-related AEs and treatment-related serious AEs were 48% and 3%, respectively. In the subgroup not previously treated with Nusinersen with two copies, the rates for independent sitting for ≥30 and ≥10 seconds were 73% and 55%, respectively; for safety outcomes, the overall rates of patients with any treatment-related AEs and treatment-related serious AEs were 51% and 6%, respectively. The rate of patients who were alive at the end of the study in the subgroup previously treated with Nusinersen was 100% in the overall population and in the subgroups of patients with two and three copies. In all the subgroups, the certainty of evidence was graded as very low for all the outcomes included.

Conclusions With very low certainty, OA showed to be a safe and efficient treatment for SMA in patients.

Authors/Disclosures
PRESENTER	No disclosure on file
Fabián A. Chávez Ecos, Sr., MD (REDECS)	Mr. Chávez Ecos has nothing to disclose.
Sandra Chavez-Malpartida	No disclosure on file
	No disclosure on file
Peggy C. Martinez Esteban, MD	Dr. Martinez Esteban has nothing to disclose.
Carlos Alva-Diaz	Carlos Alva-Diaz has nothing to disclose.

��ɫ��

��ɫ��