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心脏病学试验中的胜率:经验教训、新进展和明智的未来应用。

The win ratio in cardiology trials: lessons learnt, new developments, and wise future use.

机构信息

Medical Statistics Department, London School of Hygiene and Tropical Medicine, London, WC1E 7HT, UK.

Centre d'Investigations Cliniques Plurithématique 1433, INSERM, Université de Lorraine, Nancy, France.

出版信息

Eur Heart J. 2024 Nov 21;45(44):4684-4699. doi: 10.1093/eurheartj/ehae647.

Abstract

The win ratio method for analysing a composite clinical hierarchy of outcomes is growing in popularity especially in cardiovascular trials. This article gives a perspective on its use so far and the issues derived from that experience. Specifically, it focuses on the limitations of a conventional composite outcome; how does the win ratio work, what does it mean, and how to display its findings; guidance on choosing an appropriate clinical hierarchy of outcomes including clinical events, quantitative outcomes, and other options; the additional value of the win difference as a measure of absolute benefit: extension to stratified win ratio, subgroup analysis, matched win ratio, and covariate adjustment; determining trial size for a win ratio outcome; specific insights such as adaptive designs, use of repeat events, and use of margins and time averages for quantitative outcomes; a critique of potential misuses; availability of statistical software; and a statistical appendix on the methodological details. Throughout, each principle is illustrated by examples from specific cardiology trials. The article concludes with a set of recommendations for future use of the win ratio.

摘要

复合临床结局指标的优势比分析方法在心血管试验中越来越受欢迎。本文主要介绍了该方法的应用现状和由此产生的问题。具体来说,本文主要关注传统复合结局的局限性;优势比的工作原理、意义和展示方法;选择合适的临床结局指标的临床事件、定量结局指标和其他选择的指导意见;作为绝对获益衡量指标的赢差值的额外价值:分层优势比、亚组分析、匹配优势比和协变量调整的扩展;优势比结局的试验样本量的确定;特定的见解,如适应性设计、重复事件的使用,以及定量结局的边际和时间平均值的使用;对潜在误用的批评;统计软件的可用性;以及关于方法学细节的统计附录。贯穿全文,每个原则都通过特定的心脏病学试验的例子来说明。本文最后提出了一系列关于未来使用优势比的建议。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/34c2/11578645/26560ada1bdc/ehae647_ga.jpg

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