二元结局随机对照试验中的亚组分析：稀释与逻辑一致性性质
Subgroup analysis in randomized controlled trials with binary outcomes: dilution and logic-respecting properties
AI总结：
研究二元结局随机对照试验中亚组分析的比值比和相对响应的性质，证明比值比不适合作为疗效指标而相对响应合适，并阐明两者在逻辑一致性和稀释性质上的差异。
摘要：
亚组分析在随机对照试验中常规用于检验治疗效果在患者亚组间是否同质或由于治疗效应异质性而不同。本文研究了二元结局亚组分析中比值比和相对响应的性质，通过新的理论见解和方法学发展扩展了先前的工作。我们建立了几个新定理，描述了当两个亚组合并时，总体人群的比值比在大小和方向上如何变化。这些结果进一步证实了比值比不适合作为该亚组设置中的疗效指标，而相对响应是合适的。我们还提出了比值比和相对响应之间的正式关系，并阐明了它们在逻辑一致性性质（即总体疗效是否介于亚组疗效之间）和稀释性质（即混合亚组是否使总体比值比向1移动）方面的差异。尽管比值比通常不具有逻辑一致性，但在某些条件下它可能近似表现为具有逻辑一致性的疗效指标。为了说明我们的发现，我们基于临床试验数据给出了一个说明性示例，并讨论了其对随机对照试验中亚组分析的意义。
英文摘要：
Subgroup analysis is routinely used in randomized controlled trials to examine whether treatment effects are homogeneous across patient subgroups or differ because of treatment-effect heterogeneity. In this paper, we investigate the properties of the odds ratio and the relative response in subgroup analyses with binary outcomes, extending previous work with new theoretical insights and methodological developments. We establish several new theorems that characterize how the odds ratio for the overall population changes in both magnitude and direction when two subgroups are combined. These results further confirm that the odds ratio is inappropriate as an efficacy measure in this subgroup setting, whereas the relative response is appropriate. We also present the formal relationship between the odds ratio and the relative response, and clarify their differences in terms of the logic-respecting property, that is, whether the overall efficacy lies between the subgroup efficacies, and the dilution property, that is, whether mixing subgroups moves the overall odds ratio toward 1. Although the odds ratio is generally not logic-respecting, it may behave approximately like a logic-respecting efficacy measure under certain conditions. To illustrate our findings, we present an illustrative example based on clinical trial data and discuss its implications for subgroup analysis in randomized controlled trials.
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