脊髓性肌萎缩疾病修正药物对1型患儿通气支持需求的影响

Effect of disease-modifying drugs for spinal muscular atrophy on the ventilation support of type 1 children

目的总结脊髓性肌萎缩(SMA)疾病修正药物对1型患儿在急性呼吸衰竭后通气支持需求的影响。方法病例对照研究,纳入2020年1月至2023年7月于首都儿科研究所附属儿童医院呼吸内科因肺炎并急性呼吸衰竭住院的38例1型SMA患儿的临床特征、用药及通气支持需求情况。根据住院前是否开始并坚持使用诺西那生或利司扑兰分为治疗组和未治疗组;根据治疗组患儿出院后半年能否脱离长期持续依赖机械通气分为改善组与未改善组。组间比较采用独立样本t检验、χ^(2)检验。结果38例患儿中男女各19例,因肺炎住院年龄1.3(0.6,2.0)岁。治疗组26例、未治疗组12例,治疗组出院半年后无需长期持续依赖机械通气者比例高于未治疗组[69%(18/26)比2/12,χ^(2)=9.10,P<0.05]。改善组18例、未改善组8例,改善组首次发生急性呼吸衰竭的年龄更大[1.6(0.4,3.4)比0.5(0.3,0.7)岁,Z=2.07,P<0.05],用药时间更长[3.6(2.4,8.7)比1.2(1.2,2.4)月,t=2.74,P<0.05],合并基础疾病比例更小(1/18比6/8,χ^(2)=13.58,P<0.05)。结论SMA疾病修正药物有助于1型患儿在急性呼吸衰竭后避免长期持续依赖机械通气,坚持更长时间用药、首次发生急性呼吸衰竭年龄更大、无基础疾病者更有可能避免。

Objective To summarize the effects of disease-modifying drugs for spinal muscular atrophy(SMA)on the ventilation support of type 1 children after acute respiratory failure.Methods A case-control study was conducted,including the data of clinical characteristics,medication and ventilation supports of 38 SMA patients of type 1 with pneumonia and acute respiratory failure hospitalized in Children′s Hospital Affiliated to Capital Institute of Pediatrics from January 2020 to July 2023.Children were divided into the treatment group and the untreated group based on whether they started and persisted in using Nusinersen or Risdiplam or not before hospitalization.The differences of ventilation support between the 2 groups were analyzed.The children of the treatment group were divided into the improved group and the unimproved group based on whether they could be avoid of prolonged dependence on continuous mechanical ventilation in the next six months after discharge.The differences in clinical characteristics between the two groups were analyzed.T-test andχ^(2) test were used for comparison.Results Among the enrolled children,19 were male and 19 were female.The age was 1.3(0.6,2.0)years at the time of hospitalization due to pneumonia.There were 26 cases in the treatment group and 12 cases in the untreated group.The treatment group had a higher proportion of patients without prolonged dependence on continuous mechanical ventilation in the next six months after discharge(69%(18/26)vs.2/12,χ^(2)=9.10,P<0.05).Eighteen children were improved among the treated group,while 8 children were not.The improved group had a larger age of first onset of acute respiratory failure(1.6(0.4,3.4)vs.0.5(0.3,0.7)years,Z=2.07,P<0.05),a longer duration of medication taken before hospitalization(3.6(2.4,8.7)vs.1.2(1.2,2.4)months,t=2.74,P<0.05),and a smaller proportion with underlying diseases(1/18 vs.6/8,χ^(2)=13.58,P<0.05).Conclusions SMA disease-modifying drugs are useful for type 1 children to avoid of prolonged dependence on continuous mechanical ventilation after acute respiratory failure.The patients who take medication longer,or have acute respiratory failure for the first-time at an older age,or without underlying diseases are more likely to avoid of.