脊椎肌肉缩 尽管有新药,但死亡率:病例报告
John R Bach1, Nayara Conceição, Miguel R Goncalves
1From the Department of Physical Medicine and Rehabilitation, Rutgers University New Jersey Medical School, Newark, New Jersey (JRB); UnIC/RISE Cardiovascular R&D Unit, Faculty of Medicine, University of Porto, Porto, Portugal (NC, MRG); and Noninvasive Ventilatory Support Unit, Pulmonology Department, CAI_Vent: Home Mechanical Ventilation Program, São João University Hospital, Porto, Portugal (MRG).
脊柱肌缩 (SMA) 患者可能无法获得最佳的呼吸支持. 像机械吸气-排气这样的非侵入性方法可以预防气管切割,并改善SMA患者的治疗结果.
科学领域:
- 神经学 神经学
- 肺部病理学 肺部病理学
- 儿科 儿科 儿科
背景情况:
- 脊柱肌缩 (SMA) 1-3型患者经常经历不充分的咳流,增加急性呼吸衰竭的风险.
- 被输管治疗的SMA患者不能从呼吸机中断,通常被认为需要气管切除术.
- 这种假设可能会导致停止治疗可能挽救生命的药物,并对患者产生不良后果.
研究的目的:
- 评估非侵入性呼吸器支持的潜力,特别是机械吸气-排气 (MIE),作为医学治疗SMA型1儿童气管切除术的替代方案.
- 突出MIE的不足利用情况,尽管有证据表明其在类似患者群体中的成功率很高.
主要方法:
- 案例系列介绍了三名患有SMA型1的儿童,他们接受了医疗治疗和体力强化.
- 对所采用的呼吸支策略的审查,重点是没有尝试将输出管到非侵入性环境或MIE.
- 与有关MIE成功率的现有文献进行比较,用于输出呼吸器-不可拆卸的SMA患者的输出管.
主要成果:
- 这三个案例都涉及1型SMA的儿童接受医疗治疗,最终死亡或需要进行气管切除术.
- 在这些案例中,没有记录出对非侵入性呼吸器支持或MIE的输出试图.
- 文献表明,MIE在输出呼吸机-无法脱落,未经医疗治疗的SMA型1儿童的输出管中取得了98%以上的成功.
结论:
- 目前对1型SMA依赖于呼吸器的儿童的管理可能无法充分探索非侵入性呼吸支持选项.
- 机械吸气-排气是一种潜在地未被充分利用的,非常成功的在这个脆弱人群中排气的策略.
- 进一步调查和实施MIE可以改善呼吸道结果,并可能减少SMA患者气管切割的需要.
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