Division of Biostatistics, The University of Texas Health Science Center at Houston School of Public Health, Houston, TX, USA.
Prog Cardiovasc Dis. 2012 Jan-Feb;54(4):357-61. doi: 10.1016/j.pcad.2011.11.001.
Multiple outcomes (or multiple endpoints), such as mortality and recurrent myocardial infarction, are increasingly common in clinical trials and are often of interest in secondary analyses. Traditionally, a clinical trial protocol is built around a single event as its primary outcome, with several secondary outcomes specified, the analyses for which lack the same level of power. To accommodate all the relevant outcomes and to increase the power of the comparison in trials evaluating the efficacy of treatments for coronary heart disease, investigators often chose to construct a composite outcome. The more conventional composite outcome fails to account for the relative importance and the relationship (correlation) among its components. The purpose of this work is to demonstrate the usefulness of the Global Statistical Test, which considers the correlation between multiple outcomes, as an alternative strategy for these situations and to demonstrate its effect on hypothesis testing and power analysis issues in comparison with the traditional composite outcome analysis. Data from the cardiovascular clinical trial Systolic Hypertension in the Elderly Population are used as an example.
多种结局（或多个终点），如死亡率和再次心肌梗死，在临床试验中越来越常见，并且通常是二次分析中关注的重点。传统上，临床试验方案围绕单一事件作为主要结局构建，指定了几个次要结局，这些分析缺乏相同的效力。为了包含所有相关结局，并提高评估冠心病治疗效果的试验的比较效力，研究人员通常选择构建复合结局。较传统的复合结局未能考虑其组成部分的相对重要性和关系（相关性）。本研究的目的是展示全球统计检验的有用性，该检验考虑了多个结局之间的相关性，作为这些情况下的替代策略，并展示其在假设检验和功效分析方面与传统复合结局分析的比较效果。使用心血管临床试验 Systolic Hypertension in the Elderly Population 的数据作为示例。
