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一种用于评估具有半竞争风险的随机试验中治疗因果效应的贝叶斯非参数方法。

A Bayesian nonparametric approach for evaluating the causal effect of treatment in randomized trials with semi-competing risks.

作者信息

Xu Yanxun, Scharfstein Daniel, Müller Peter, Daniels Michael

机构信息

Department of Applied Mathematics and Statistics, Johns Hopkins University, 3400 N. Charles Street, Baltimore, MD 21218, USA

Department of Biostatistics, Johns Hopkins University, 615 N Wolfe St, Baltimore, MD 21205, USA.

出版信息

Biostatistics. 2022 Jan 13;23(1):34-49. doi: 10.1093/biostatistics/kxaa008.

DOI:10.1093/biostatistics/kxaa008
PMID:32247284
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10448950/
Abstract

We develop a Bayesian nonparametric (BNP) approach to evaluate the causal effect of treatment in a randomized trial where a nonterminal event may be censored by a terminal event, but not vice versa (i.e., semi-competing risks). Based on the idea of principal stratification, we define a novel estimand for the causal effect of treatment on the nonterminal event. We introduce identification assumptions, indexed by a sensitivity parameter, and show how to draw inference using our BNP approach. We conduct simulation studies and illustrate our methodology using data from a brain cancer trial. The R code implementing our model and algorithm is available for download at https://github.com/YanxunXu/BaySemiCompeting.

摘要

我们开发了一种贝叶斯非参数(BNP)方法,用于评估随机试验中治疗的因果效应,其中非终末事件可能被终末事件截尾,但反之则不然(即半竞争风险)。基于主分层的思想,我们定义了一种用于治疗对非终末事件因果效应的新估计量。我们引入了由一个敏感性参数索引的识别假设,并展示了如何使用我们的BNP方法进行推断。我们进行了模拟研究,并使用来自一项脑癌试验的数据说明了我们的方法。实现我们模型和算法的R代码可在https://github.com/YanxunXu/BaySemiCompeting下载。

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