Use of a global test for multiple outcomes in stroke trials with application to the national institute of neurological disorders and stroke t-PA stroke trial
Use of a global test for multiple outcomes in stroke trials with application to the national institute of neurological disorders and stroke t-PA stroke trial
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DOI：
10.1161/01.str.27.11.2136
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发表时间：
1996-11-01
期刊：
影响因子：
8.3
通讯作者：
Grotta, J
中科院分区：
文献类型：
--
作者：
Tilley, BC;Marler, J;Grotta, J
国家神经疾病和卒中研究所（NINDS）举办了一次研讨会，讨论分析具有多种预定结局的急性卒中试验的统计方法，目的是计划对NINDS t-PA卒中试验进行统计分析，该试验是一项重组组织纤溶酶原激活剂（rt-PA）治疗急性缺血性卒中患者的随机、双盲、安慰剂对照试验。治疗成功定义为在Barthel指数、改良兰金量表、格拉斯哥结局量表上获得良好结局的患者比例的“一致和有说服力的差异”。和国立卫生研究院卒中量表。试验的数据和安全性监测委员会推荐了这一结果，因为该委员会不认为单一结果的阳性结果将提供足够的疗效证据。评论摘要研讨会参与者接受全球测试作为检验主要试验假设的可行方法。临床医生参与者主张将结局分类为有利/不利，对于评价具有潜在严重副作用的药物，结局比连续结局更具临床意义。他们一致认为，当没有单一结果被接受时，全球测试适合缺血性卒中。假设性的特例例子表明，高度相关的结果削弱了全球检验的效力。NINDS的t-PA中风试验数据表明全球test.Conclusions的临床interpretability研讨会的参与者得出结论，全球统计数据应用于测试试验的主要假设伴随着二次测试的个人成果。讲习班与会者建议让临床/科学界熟悉全球办法。
Background The National Institute of Neurological Disorders and Stroke (NINDS) held a workshop on statistical approaches to analysis of acute stroke trials that have multiple prespecified outcomes, An objective was to plan for statistical analysis of the NINDS t-PA Stroke Trial, a randomized, double-blind, placebo-controlled trial of recombinant tissue plasminogen activator (rt-PA) for patients with acute ischemic stroke. Treatment success was defined as a ''consistent and persuasive difference'' in the proportion of patients achieving favorable outcomes on the Barthel Index, Modified Rankin Scale, Glasgow Outcome Scale. and National Institutes of Health Stroke Scale. The Data and Safety Monitoring Committee for the trial recommended this outcome because the committee did not believe that a positive result for a single outcome would provide sufficient evidence of efficacy.Summary of Comment Workshop participants accepted the global test as a viable approach to testing the primary trial hypothesis. Clinician participants advocated categorizing outcomes as favorable/unfavsrable, outcomes more clinically meaningful than continuous outcomes for evaluating a drug with potentially serious side effects. They agreed that a global test was appropriate for ischemic stroke when no single outcome is accepted. Hypothetical, special-case examples illustrate that highly correlated outcomes diminish the power of the global test. NINDS t-PA Stroke Trial data demonstrate the clinical interpretability of the global test.Conclusions Workshop participants concluded that a global statistic should be used to test the trial's primary hypothesis accompanied by secondary tests of individual outcomes. Workshop participants recommended familiarizing the clinical/scientific community with the global approach.