Department of Clinical Epidemiology and Biostatistics, McMaster University, 1280 Main Street West, Hamilton, Ontario L8S4L8, Canada; Division of Cardiology, Department of Internal Medicine, Veterans Affairs Medical Center, 3495 Bailey Avenue, Buffalo, NY 14215, USA.
Department of Clinical Epidemiology and Biostatistics, McMaster University, 1280 Main Street West, Hamilton, Ontario L8S4L8, Canada; Division of Gastroenterology, Department of Internal Medicine, McMaster University, 280 Main Street West, Hamilton, Ontario L8S4L8, Canada.
J Clin Epidemiol. 2017 Feb;82:4-11. doi: 10.1016/j.jclinepi.2016.12.001. Epub 2016 Dec 11.
Composite end points are frequently used in reports of clinical trials. One rationale for the use of composite end points is to account for competing risks. In the presence of competing risks, the event rate of a specific event depends on the rates of other competing events. One proposed solution is to include all important competing events in one composite end point. Clinical trialists require guidance regarding when this approach is appropriate.
To identify publications describing criteria for use of composite end points for competing risk and to offer guidance regarding when a composite end point is appropriate on the basis of competing risks.
METHODS, DATA SOURCES, STUDY SELECTION AND DATA EXTRACTION: We searched MEDLINE, CINAHL, EMBASE, The Cochrane's Central & Systematic Review databases including the Health Technology Assessment database, and the Cochrane's Methodology register from inception to April 2015, and candidate textbooks, to identify all articles providing guidance on this issue. Eligible publications explicitly addressed the issue of a composite outcome to address competing risks. Two reviewers independently screened the titles and abstracts for full-text review; independently reviewed full-text publications; and abstracted specific criteria authors offered for use of composite end points to address competing risks.
Of 63,645 titles and abstracts, 166 proved potentially relevant of which 43 publications were included in the final review. Most publications note competing risks as a reason for using composite end points without further elaboration. None of the articles or textbook chapters provide specific criteria for use of composite end points for competing risk. Some advocate using composite end points to avoid bias due to competing risks and others suggest that composite end points seldom or never be used for this purpose. We recommend using composite end points for competing risks only if the competing risk is plausible and if it occurs with sufficiently high frequency to influence the interpretation of the effect of intervention on the end point of interest. These criteria will seldom be met. Review of heart failure trials published in the New England Journal of Medicine revealed that many of them use the composite end point of death or hospitalization; none of the trials, however, satisfied our criteria.
The existing literature fails to provide clear guidance regarding use of composite end point for competing risks. We recommend using composite end points for competing risks only if the competing risk is plausible and if it occurs sufficiently often.
复合终点在临床试验报告中经常被使用。使用复合终点的一个理由是为了考虑竞争风险。在存在竞争风险的情况下，特定事件的事件发生率取决于其他竞争事件的发生率。一种建议的解决方案是将所有重要的竞争事件包含在一个复合终点中。临床研究者需要关于何时使用这种方法的指导。
确定描述使用复合终点来处理竞争风险的标准的出版物，并根据竞争风险提供何时使用复合终点的指导。
方法、数据来源、研究选择和数据提取：我们从开始到 2015 年 4 月，在 MEDLINE、CINAHL、EMBASE、Cochrane 中央和系统评价数据库（包括卫生技术评估数据库）和 Cochrane 方法学登记处进行了搜索，以及候选教科书，以确定所有提供关于这个问题的指导的出版物。合格的出版物明确地解决了处理竞争风险的复合结果问题。两位审查员独立筛选标题和摘要进行全文审查；独立审查全文出版物；并提取作者提供的用于处理竞争风险的复合终点使用的具体标准。
在 63645 个标题和摘要中，有 166 个被证明是潜在相关的，其中 43 篇出版物被纳入最终审查。大多数出版物指出竞争风险是使用复合终点的原因，而没有进一步阐述。没有一篇文章或教科书章节提供了用于处理竞争风险的复合终点的具体标准。有些主张使用复合终点来避免因竞争风险引起的偏倚，而另一些则认为复合终点很少或从不用于此目的。我们建议仅在竞争风险合理且足以影响对感兴趣的终点的干预效果的解释时，才使用竞争风险的复合终点。这些标准很少得到满足。对发表在《新英格兰医学杂志》上的心力衰竭试验的审查表明，其中许多试验使用死亡或住院的复合终点；然而，没有一个试验符合我们的标准。
现有文献未能就竞争风险的复合终点使用提供明确的指导。我们建议仅在竞争风险合理且经常发生时才使用竞争风险的复合终点。
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