Chinese Journal of Evidence-Based Pediatrics ›› 2022, Vol. 17 ›› Issue (6): 420-425.DOI: 10.3969/j.issn.1673-5501.2022.06.003

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Natural history of spinal muscular atrophy: A systematic review and meta-analysis

HU Chaoping1a, LI Wenhui1a, ZHU Xiaomei1a, LI Yijie1a, WANG Huishan1b, ZHOU Shuizhen1a,WANG Yi1a,2, ZHANG Chongfan1b   

  1. 1 Children's Hospital of Fudan University, Shanghai 201102, China, a. Department of Neurology,  b. GRADE Center; 2 Hospital Management Office of Fudan University, Shanghai 200032, China
  • Received:2022-12-20 Revised:2022-12-21 Online:2022-12-25 Published:2022-12-25
  • Contact: WANG Yi; ZHANG Chongfan

Abstract: Objective: To systematically analyze the survival rate and trajectory of motor function in the natural history of SMA patients so as to provide benchmark data for the intervention of SMA. Methods: PubMed, Embase, Cochrane, CNKI, CBM and Wanfang database were searched by using the keywords of spinal muscular atrophy and natural history, from the inception to November 27th 2022. Results: A total of 17 case series reports (n=1905) and 2 sham controls of Nusinersen in RCTs (n=83) for SMA were included in the analysis. The survival rates at the age of 6 months (4 studies), 12 months (5 studies) and 18 months (4 studies) were 86%, 52% and 32%, respectively. The meta-analysis of event-free survival rates from natural history case series reports of type 1 SMA showed that it was 76%,34% and 16% at the age of 6 months (3 studies), 12 months (4 studies) and 18 months (3 studies) respectively. Conclusion: Natural history of SMA patients varied in different types, of which type 1 SMA showed lower survival and event-free survival rates, while type 2-3 SMA patients present with slower regression of motor function.

Key words: Spinal muscular atrophy, Natural history, Survival rate, Motor function