Abstract:

Objective Meta-analysis was used to evaluate clinical efficacy in children with spinal muscular atrophy treated with nusinersen. Methods Cochrane Library, Embase, PubMed, Web of Science, ClinicalTrial.gov, CBM, CNKI, Wanfang and VIP databases were searched from inception to December 31, 2022. Stata 14.0 was used for meta-analysis after two researchers independently screened the literature, extracted data, and evaluated the risk of bias in the included studies. Results A total of 14 cohort studies were included, involving 1274 SMA patients <18 years of age. During therapy, motor function indicators showed significant improvement in the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), Hammersmith Infant Neurological Examination Part 2 (HINE-2) score, Hammersmith Functional Motor Scale Expanded (HFMSE) and Revised Upper Limb Module (RULM) score, and combined mean differences were 9.08(95%CI: 7.17-10.99), 2.08(95%CI: 1.26-2.90), 3.83(95%CI: 2.15-5.50) and 2.42(95%CI: 1.33-3.52), respectively. The clinical improvement rates were 0.77(95%CI: 0.71-0.82), 0.39(95%CI: 0.31-0.48), 0.53(95%CI: 0.33-0.72), and 0.56(95%CI: 0.49-0.63), respectively. Conclusions The motor function of children with SMA type 1, 2, and 3 was significantly improved by nusinersen. Limited by the quality and sample size of the included studies, the above conclusions need to be verified by more high-quality studies.

Key words: spinal muscular atrophy, nusinersen, motor function, meta-analysis, child