OBJECTIVE To evaluate respiratory support, nutritional support and safety in children with spinal muscular atrophy(SMA) treated with nusinersen by meta-analysis, to provide evidence-based evidence for clinical treatment of SMA.
METHODS Cochrane Library, Embase, PubMed, Web of Science, ClinicalTrial.gov, CBM, CNKI, Wanfang and VIP databases were searched from inception to December 31, 2022. After extracting data and evaluating literature quality, RevMan 5.4 software was used for meta-analysis.
RESULTS A total of 11 cohort studies were included, and involving 1153 SMA patients(<18 years), th results of meta-analysis indicated that during nusinersen treatment, motor function indicators showed significant improvements in children with SMA, WHO motor milestone improvement rate was 0.2695%CI(0.20, 0.32), P<0.00001, but respiratory support and nutritional support were not positively affected, and the overall incidence of adverse events(occurrence numbers/total number of persons) was 0.3195%CI(0.19, 0.41), P<0.00001, death rate was 0.0395%CI(0.01, 0.06), P=0.04, treatment with drawal rate was 0.0495%CI(0.03, 0.06), P=0.03; among the adverse reactions, the proportion of severe adverse reactions(occurrence cases/total cases) was 0.6595%CI(0.44, 0.86), P<0.00001. The main adverse events were respiratory tract infection, lumbar puncture syndrome, abdominal symptoms, fever, acute respiratory failure, other infections, other adverse events such as fractures, seizures, cerebrospinal fluid leakage, weakness.
CONCLUSION While improvements in motor function, a positive effect of nusinersen treatment on the need for ventilator support or nutritional support in children with SMA type 1, 2 and 3 was not observe , and using nusinersen has higher adverse reactions in children. However, the sample size of the current studies is small, all observational studies, and the evidence is low-quality, more high-quality studies were still necessary to confirm the result.
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