试验设计的有效统计分析:复合结果的胜率和相关方法。

IF 2 3区 医学 Q2 ANESTHESIOLOGY
Wilson Fandino, Matthew Dodd, Gudrun Kunst, Tim Clayton
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引用次数: 0

摘要

在随机对照临床试验中,复合结局常用于研究治疗效果。这种方法很受欢迎,因为它增加了观察事件的数量,增强了统计能力,同时减少了所需的患者样本量。然而,综合结果并不能深入了解单个终点的影响。当死亡率与不太关键但临床相关的终点相结合时,或者当单个终点的临床重要性差异显著时,这一点尤为重要。因此,解释复合结果可能具有挑战性。这篇叙述性综述介绍了胜率(WR),这是一种在复合结果中优先考虑单个终点的方法。WR通过考虑每个组成部分的临床重要性,优先考虑最关键的终点,如死亡,而不是不太重要的事件,提供了一种替代复合结局的方法。尽管WR在心血管试验中很受欢迎,但这种方法尚未广泛应用于其他临床研究领域。我们认为,围手术期和围手术期研究人员可以考虑WR和相关的方法,当感兴趣的结果不具有类似的临床重要性。为此,了解WR的优点和局限性对于利用其优点至关重要,同时避免对该技术的潜在滥用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Efficient statistical analysis of trial designs: win ratio and related approaches for composite outcomes.

In randomized controlled clinical trials, composite outcomes are often used to study treatment effects. This approach is popular because it increases the number of observed events, enhancing statistical power while reducing the required patient sample size. However, composite outcomes do not provide insight into the effect of individual endpoints. This becomes particularly relevant when mortality is combined with less critical but clinically relevant endpoints or when the clinical importance of individual endpoints varies significantly. As a result, interpreting composite outcomes can be challenging.This narrative review introduces the win ratio (WR), a method for prioritizing individual endpoints within a composite outcome. The WR offers an alternative to composite outcomes by considering the clinical importance of each component and prioritizing the most critical endpoint, such as death, over less significant events.Despite the popularity of the WR among cardiovascular trialists, this approach has not been extensively used in other areas of clinical research. We contend, that perioperative and periprocedural researchers could consider the WR and related approaches when the outcomes of interest are not of similar clinical importance. To this end, understanding the benefits and limitations of the WR will be essential to exploit its benefits, while avoiding potential misuses of the technique.

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