onasemnogene abeparvovec基因治疗脊髓性肌萎缩症的安全性和有效性：一项系统评价和荟萃分析。
Safety and efficacy of gene therapy with onasemnogene abeparvovec in the treatment of spinal muscular atrophy: A systematic review and meta-analysis.
作者信息
Yang Dongling, Ruan Yiyan, Chen Yuyi
机构信息
Ruikang Clinical Medical College, Guangxi University of Chinese Medicine, Nanning, Guangxi, China.
Department of Pediatric Neurology, Maternal and Child Health Hospital of Guangxi Zhuang Autonomous Region, Guangxi Clinical Research Center for Pediatric Diseases, Nanning, Guangxi, China.
出版信息
J Paediatr Child Health. 2023 Mar;59(3):431-438. doi: 10.1111/jpc.16340. Epub 2023 Feb 1.
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: 65.890.5) and elevated aminotransferases (71.7%, 95%CI: 62.580.1) 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.
脊髓性肌萎缩症（SMA）是一种常染色体隐性遗传性疾病，可导致进行性肌肉无力和萎缩。我们的系统评价和荟萃分析旨在探讨onasemnogene abeparvovec对SMA患者的疗效和安全性。我们检索了截至2022年4月的PubMed、EMBASE、科学网和考科蓝数据库。纳入了10篇报告，共250例SMA患者。在短期和长期随访中均检测到CHOP INTEND和运动里程碑有显著改善。常见的不良事件包括发热、呕吐、血小板减少和转氨酶升高。血小板减少（79.3%，95%CI：65.890.5）和转氨酶升高（71.7%，95%CI：62.580.1）在8个月以上的SMA患者中更为常见。尽管缺乏随机对照试验数据，且证明onasemnogene abeparvovec治疗SMA安全性和有效性的证据质量较低，但数据表明，它对患有这种疾病的患者是一个有价值的选择。
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