作者@article {van den Berge1610 ={伦纳德h . van den Berg埃里克·索伦森和加里Gronseth埃里克·a·Macklin Jinsy安德鲁斯和罗伯特·h·Baloh迈克尔·贝纳塔尔和詹姆斯·d·贝瑞和阿德里亚诺亚蔡Philippe Corcia和安琪拉其全称天使爱美丽k Gubitz和凯瑟琳Lomen-Hoerth克里斯托弗·j·麦克德莫特和埃里克·p·Pioro杰弗里·罗森菲尔德和Vincenzo Silani和马丁·r·特纳和马库斯·韦伯和本杰明·里克斯布鲁克斯和罗伯特·g·米勒和Hiroshi Mitsumoto和艾利房子ALS临床试验指导集团},title ={修订艾利房子共识肌萎缩性侧索硬化症的临床试验设计和实现指南},体积={92}={14},页面= {e1610——e1623} = {2019}, doi = {10.1212 / WNL。出版商0000000000007242}= {Wolters Kluwer健康,公司代表美国神经病学学会},文摘={目的修改1999艾利共识指南的设计和实现临床前研究和临床试验治疗肌萎缩性脊髓侧首页索硬化症(ALS)。方法达成共识委员会由140国际ALS的关键成员社区(肌萎缩性侧索硬化症研究人员,临床医生、病人的代表,研究经费,代表行业和监管机构)解决9需要在ALS的研究领域:(1)临床前研究;(2)生物和表型异质性;(3)结果评价;(4)疾病和症状的干预措施;(5)招聘和保留;(6)生物标志物;(7)临床试验阶段;(8)超越传统的试验设计;和(9)统计方面的考虑。分配给1 8个部分组成,委员会成员生成一系列草案指南基于{\ textquotedblright} {\ textquotedblleft}背景的(前)临床问题和一个{\ textquotedblright} {\ textquotedblleft}原理概述了证据和专家意见。 Following a 2-day, face-to-face workshop at the Airlie House Conference Center, a modified Delphi process was used to develop draft consensus research guidelines, which were subsequently reviewed and modified based on comments from the public. Statistical experts drafted a separate document of statistical considerations (section 9).Results In this report, we summarize 112 guidelines and their associated backgrounds and rationales. The full list of guidelines, the statistical considerations, and a glossary of terms can be found in data available from Dryad (appendices e-3{\textendash}e-5, doi.org/10.5061/dryad.32q9q5d). The authors prioritized 15 guidelines with the greatest potential to improve ALS clinical research.Conclusion The revised Airlie House ALS Clinical Trials Consensus Guidelines should serve to improve clinical trial design and accelerate the development of effective treatments for patients with ALS.ALS=amyotrophic lateral sclerosis; NIV=noninvasive ventilation; PALS=people living with amyotrophic lateral sclerosis; QOL=quality of life}, issn = {0028-3878}, URL = {//www.ez-admanager.com/content/92/14/e1610}, eprint = {//www.ez-admanager.com/content/92/14/e1610.full.pdf}, journal = {Neurology} }