Yang Siyun, Zhou Ruiwen, Li Fan, Thomas Laine E
Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, NC, USA.
Division of Biostatistics, Washington University in St. Louis, Missouri, USA.
Stat Methods Med Res. 2023 Oct;32(10):1919-1935. doi: 10.1177/09622802231188517. Epub 2023 Aug 9.
Evaluating causal effects of an intervention in pre-specified subgroups is a standard goal in comparative effectiveness research. Despite recent advancements in causal subgroup analysis, research on time-to-event outcomes has been lacking. This article investigates the propensity score weighting method for causal subgroup survival analysis. We introduce two causal estimands, the subgroup marginal hazard ratio and subgroup restricted average causal effect, and provide corresponding propensity score weighting estimators. We analytically established that the bias of subgroup-restricted average causal effect is determined by subgroup covariate balance. Using extensive simulations, we compare the performance of various combinations of propensity score models (logistic regression, random forests, least absolute shrinkage and selection operator, and generalized boosted models) and weighting schemes (inverse probability weighting, and overlap weighting) for estimating the causal estimands. We find that the logistic model with subgroup-covariate interactions selected by least absolute shrinkage and selection operator consistently outperforms other propensity score models. Also, overlap weighting generally outperforms inverse probability weighting in terms of balance, bias and variance, and the advantage is particularly pronounced in small subgroups and/or in the presence of poor overlap. We applied the methods to the observational Comparing Options for Management: PAtient-centered REsults for Uterine Fibroids study to evaluate the causal effects of myomectomy versus hysterectomy on the time to disease recurrence in a number of pre-specified subgroups of patients with uterine fibroids.
评估预先指定亚组中干预措施的因果效应是比较效果研究的一个标准目标。尽管因果亚组分析最近取得了进展,但针对事件发生时间结局的研究仍很缺乏。本文研究了用于因果亚组生存分析的倾向评分加权方法。我们引入了两个因果估计量,即亚组边际风险比和亚组受限平均因果效应,并提供了相应的倾向评分加权估计量。我们通过分析确定,亚组受限平均因果效应的偏差由亚组协变量平衡决定。通过广泛的模拟,我们比较了倾向评分模型(逻辑回归、随机森林、最小绝对收缩和选择算子以及广义增强模型)和加权方案(逆概率加权和重叠加权)的各种组合在估计因果估计量方面的性能。我们发现,由最小绝对收缩和选择算子选择的具有亚组 - 协变量交互作用的逻辑模型始终优于其他倾向评分模型。此外,在平衡、偏差和方差方面,重叠加权通常优于逆概率加权,并且在小亚组和/或重叠较差的情况下优势尤为明显。我们将这些方法应用于观察性的“管理比较选项:子宫肌瘤患者为中心的结果”研究,以评估子宫肌瘤患者多个预先指定亚组中肌瘤切除术与子宫切除术对疾病复发时间的因果效应。