Consensus statement for Standard of Care in Spinal Muscular Atrophy
Consensus statement for Standard of Care in Spinal Muscular Atrophy
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DOI：
10.1177/0883073807305788
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发表时间：
2007-08-01
影响因子：
1.9
通讯作者：
Trela, Anthony
中科院分区：
文献类型：
--
作者：
Wang, Ching H.;Finkel, Richard S.;Trela, Anthony
脊髓性肌萎缩症是一种神经退行性疾病，需要多学科的医疗护理。近年来，对脊髓性肌萎缩症分子发病机制的了解和医学技术的进步，在治疗脊髓性肌萎缩症患者方面没有取得类似的进展。医疗实践的差异，加上家庭资源和价值观的差异，导致了不同的临床结果，这可能会影响在临床试验期间对治疗效果的有效衡量。脊髓性肌萎缩症国际护理标准委员会成立于2005年，目的是为这些患者的临床护理建立实践指南。12名核心委员会成员通过电话会议、电子邮件通信、德尔福调查和2次面对面会议，与该领域的60多名脊肌萎缩症专家合作，就5个护理领域达成共识：诊断/新干预、肺、胃肠/营养、整形外科/康复和姑息治疗。就脊髓性肌萎缩症的常见医疗问题、诊断战略、评估和监测建议以及每个护理领域的治疗干预措施等几个主题达成了共识。起草了一份共识声明，根据患者的3个功能级别：非保姆、保姆和助行者，解决了5个护理领域。该委员会还发现了几种缺乏共识的医疗做法，需要进一步调查。作者的意图是将本文件用作指导方针，而不是作为其护理的实践标准。迫切需要一个脊髓性肌萎缩症的实践标准，以帮助这些患者的多学科护理。
Spinal muscular atrophy is a neurodegenerative disease that requires multidisciplinary medical care. Recent progress in the understanding Of Molecular pathogenesis of spinal muscular atrophy and advances in medical technology have not been matched by similar developments in the care for spinal muscular atrophy patients. Variations in medical practice coupled with differences in family resources and values have resulted in variable clinical outcomes that are likely to compromise valid measure of treatment effects during clinical trials. The International Standard of Care Committee for Spinal Muscular Atrophy was formed in 2005, with a goal of establishing practice guidelines for clinical care of these patients. The 12 core committee members worked with more than 60 spinal muscular atrophy experts in the field through conference calls, e-mail communications, a Delphi survey, and 2 in-person meetings to achieve consensus on 5 care areas: diagnostic/new interventions, Pulmonary, gastrointestinal/nutrition, orthopedics/rehabilitation, and palliative care. Consensus was achieved on several topics related to common medical problems in Spinal Muscular atrophy, diagnostic strategies, recommendations for assessment and monitoring, and therapeutic interventions in each care area. A consensus statement was drafted to address the 5 care areas according to 3 functional levels of the patients: nonsitter, sitter, and walker. The committee also identified several medical practices lacking consensus and warranting further investigation. It is the authors' intention that this document be used as a guideline, not as a practice standard for their care. A practice standard for spinal muscular atrophy is urgently needed to help with the multidisciplinary care of these patients.