Composite outcomes in randomized trials - Greater precision but with greater uncertainty?
Composite outcomes in randomized trials - Greater precision but with greater uncertainty?
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DOI：
10.1001/jama.289.19.2554
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发表时间：
2003-05-21
影响因子：
120.7
通讯作者：
Griffin, C
中科院分区：
文献类型：
--
作者：
Freemantle, N;Calvert, M;Griffin, C
在随机试验中，将多个终点组合在一起的复合结果经常被用作主要结果衡量标准，而且往往与统计效率的提高有关。然而，事实可能证明，这些措施对解释结果具有挑战性。在这篇文章中，我们检查了在主要临床试验中复合结果的使用，评估了支持和反对它们的论点，并就它们的应用和报告提供了指导。为了评估报告的发生率和质量，我们采用敏感的搜索策略，系统地回顾了1997-2001年间《内科年鉴》、《BMJ》、《循环》、《临床传染病》、《美国心脏病学会杂志》、《美国医学会杂志》、《柳叶刀》、《新英格兰医学杂志》和《中风》中复合终点的使用情况。我们选择了167份随机试验的原始报告(总共有300276名患者)进行审查，这些报告包括了包含所有原因死亡率的综合主要结果。63项试验(38%)对于主要终点和死亡率部分都是中性的。63项试验(36%)报告了主要结果指标的显著结果，但对死亡率部分没有影响。只有6项试验(4%)对死亡率部分有意义，但对主要综合结果没有影响，而19项试验(11%)对两者都有意义。22项试验(13%)没有充分报道。我们的审查表明，对综合结果的报告通常是不充分的，这意味着结果适用于综合结果的个别组成部分，而不仅仅是整个综合结果。目前关于开展和报告临床试验的指南可以修订，以反映临床试验中综合结果的普遍使用。
Composite outcomes, in which multiple end points are combined, are frequently used as primary outcome measures in randomized trials and are often associated with increased statistical efficiency. However, such measures may prove challenging for the interpretation of results. In this article, we examine the use of composite outcomes in major clinical trials, assess the arguments for and against them, and provide guidance on their application and reporting. To assess incidence and quality of reporting, we Systematically reviewed the use of composite end points in clinical trials in Annals of Internal Medicine, BMJ, Circulation, Clinical Infectious Diseases, Journal of the American College of Cardiology, JAMA, Lancet, New England Journal of Medicine, and Stroke from 1997 through 2001 using a sensitive search strategy. We selected for review 167 original reports of randomized trials (with a total of 300276 patients) that included a composite primary outcome that incorporated all-cause mortality. Sixty-three trials (38%) were neutral both for the primary end point and the mortality component Sixty trials (36%) reported significant results for the primary outcome measure but not for the mortality component Only 6 trials (4%) were significant for the mortality component but not for the primary composite outcome, whereas 19 trials (11%) were significant for both. Twenty-two trials (13%) were inadequately reported. Our review suggests that reporting of composite outcomes is generally inadequate, implying that the results apply to the individual components of the composite outcome rather than only to the overall composite. Current guidelines for the undertaking and reporting of clinical trials could be revised to reflect the common use of composite outcomes in clinical trials.