Safety and efficacy of gene therapy with onasemnogene abeparvovec in the treatment of spinal muscular atrophy: A systematic review and meta-analysis.

Abstract

Spinal muscular atrophy (SMA) is an autosomal recessive hereditary disease which leads to progressive muscle weakness and atrophy. Our systematic review and meta-analysis aims to explore the efficacy and safety of onasemnogene abeparvovec in SMA patients. We searched PubMed, EMBASE, Web of Science and Cochrane through April 2022. Ten reports enrolling 250 SMA patients were included. CHOP INTEND and motor-milestone significant improvements were detected at both short- and long-term follow-up. Common adverse events included pyrexia, vomiting, thrombocytopenia and elevated aminotransferases. Thrombocytopenia (79.3%, 95%CI: 65.8~90.5) and elevated aminotransferases (71.7%, 95%CI: 62.5~80.1) were more common in SMA patients aged older than 8 months. Despite the paucity of randomized control trial data and low quality of evidence to establish the safety and efficacy of onasemnogene abeparvovec in the treatment of SMA, the data suggest that it is a valuable option for patients with this condition.

• Authors: Dongling Yang, Yiyan Ruan, Yuyi Chen
• Journal: Journal of paediatrics and child health (J Paediatr Child Health) Vol. 59 Issue 3 Pg. 431-438 (03 2023) ISSN: 1440-1754 [Electronic] Australia
• PMID: 36722610 (Publication Type: Meta-Analysis, Systematic Review, Journal Article, Review, Research Support, Non-U.S. Gov't)
• Copyright: © 2023 The Authors. Journal of Paediatrics and Child Health published by John Wiley & Sons Australia, Ltd on behalf of Paediatrics and Child Health Division (The Royal Australasian College of Physicians).

Chemical References

• Transaminases

Topics

• Humans
• Aged
• Spinal Muscular Atrophies of Childhood
• Muscular Atrophy, Spinal (genetics, therapy)
• Genetic Therapy
• Thrombocytopenia
• Transaminases