摘要目的 探讨无创通气治疗脊髓性肌萎缩(SMA)患儿睡眠呼吸紊乱的有效性和长期作用.方法 前瞻性研究2016年3月至2018年1月在首都儿科研究所附属儿童医院呼吸内科就诊,有中重度睡眠呼吸紊乱的17例SMA患儿的夜间无创通气的治疗效果.患儿分为治疗组(使用无创通气)9例和对照组(未使用无创通气),8例拟行无创通气治疗的患儿需完成睡眠呼吸压力滴定.治疗组和对照组患儿均随访1年.采用配对样本t检验,比较完成睡眠呼吸压力滴定的SMA患儿无创通气治疗前后多道睡眠描记术中各参数的变化.采用Mann-Whitney U秩和检验,比较治疗组和对照组患儿1年内发生呼吸道感染频率的差异.结果 17例SMA患儿男10例、女7例,就诊时年龄(5.1±2.9)岁.8例患儿完成睡眠呼吸压力滴定后,多道睡眠描记术结果显示,呼吸暂停低通气指数为(3.8±2.5)次/h,与滴定前(16.6±9.7)次/h相比显著减低(t=4.086,P=0.005),滴定后低通气指数为(2.4±1.2)次/h,与滴定前(7.2±4.7)次/h相比显著减低(t=2.779,P=0.027),滴定后平均血氧饱和度为0.966±0.007,与滴定前0.946±0.015相比有显著改善(t=-5.292,P=0.001),滴定后最低血氧饱和度为0.906±0.023,与滴定前0.786±0.092相比有显著改善(t=-3.938,P=0.006).治疗组9例长期家庭夜间无创通气患儿呼吸机模式均为双水平气道正压通气S/T模式,吸气相气道正压8～14 cmH2O(1 cmH20=0.098 kPa),呼气相气道正压4～6 cmH2O,1年后患上呼吸道感染1.0(0,3.0)次/年,明显低于8例对照组未使用无创通气患儿的3.0(2.3,7.0)次/年(Z=-2.245,P=0.023),治疗后下呼吸道感染0(0,0)次/年,明显低于对照组的2.0(1.3,4.5)次/年(Z=-3.189,P=0.001),治疗后住院频率0(0,0)次/年,明显低于对照组的1.0(1.0,4.3)次/年(Z=-3.420,P＜0.01),治疗后重症监护室住院频率0(0,0)次/年,明显低于对照组的0.5(0,1.0)次/年(Z=-3.353,P=0.029).结论 无创通气可以有效治疗SMA患儿睡眠呼吸紊乱,且长期进行夜间无创通气治疗可减少SMA患儿呼吸道感染的发生.

abstractsObjective To study the short-term and long-term efficacy of the non-invasive ventilation treatment in children with spinal muscular atrophy (SMA) and sleep-disordered breathing.Methods This was a prospective research to study the effect of night-time non-invasive ventilation in children with SMA and moderate to severe sleep-disordered breathing during March 2016 to January 2018,from the Pulmonary Department of Capital Institute of Pediatrics Affiliated Children's Hospital.Patients were divided into the treated group (with night-time non-invasive ventilation) and the control group (without ventilator).Sleep breathing pressure titration was suggested to the patients who were prepared to receive non-invasive ventilation.All cases were followed up for one year.Parameters'changes in polysomnography were assessed (paired t-test) in titration patients.Frequency of respiratory tract infection during the next year in the patients with and without ventilation was collected and compared (Mann-Whitney U-test).Results Seventeen cases were recruited.The average age was (5.1±2.9) years,10 cases were boys and 7 cases were girls.In the titration group (8 patients),after non-invasive ventilation,the average apnea hypopnea index was (3.8±2.5) times/h (t=4.086,P=0.005),hypopnea index was (2.4± 1.2) times/h (t=2.779,P=0.027),average oxygen saturation during total sleep time was 0.966±0.007 (t=-5.292,P=0.001),and the minimum oxygen saturation was 0.906±0.023 (t=-3.938,P=0.006).All the above parameters were significantly improved after treatment.Than before,which was (16.6±9.7) times/h,(7.2±4.7) times/h,0.946±0.015,0.786±0.092 respectively.Ventilator mode for the 9 children with long time non-invasive ventilation at home was Bi-level positive airway pressure S/T.The positive airway pressure was set at 8-14 cmH20 (1 cmH2O=0.098 kPa) in inspiratory phase and 4-6 cmH20 in expiratory phase.In the treated group (9 patients),the average frequency of upper respiratory tract infection was 1.0 (0,3.0) times/year (Z=-2.245,P=0.023),the lower respiratory tract infection was 0 (0,0) times/year (Z=-3.189,P=0.001),hospitalization was 0 (0,0) times/year (Z=-3.420,P＜0.01),and admission to intensive care unit was 0 (0,0) times/year (Z=-3.353,P=0.029).All the above indexes were significantly decreased compared with the control group (8 patients),which was 3.0 (2.3,7.0) times/year,2.0 (1.3,4.5) times/year,1.0 (1.0,4.3) times/year,0.5 (0,1.0) times/year respectively.Conclusion Non-invasive ventilation is efficient to SMA children with sleep-disordered breathing,and also can reduce the incidence of respiratory tract infections for children with SMA.