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具有事件时间数据的混合控制分析的案例加权功效先验。

Case weighted power priors for hybrid control analyses with time-to-event data.

机构信息

Department of Biostatistics and Data Science, The University of Texas Health Science Center at Houston, 1200 Pressler St, Houston, TX 77030, USA.

Department of Biostatistics, Genentech,  South San Francisco, CA 94080, USA.

出版信息

Biometrics. 2024 Mar 27;80(2). doi: 10.1093/biomtc/ujae019.

Abstract

We develop a method for hybrid analyses that uses external controls to augment internal control arms in randomized controlled trials (RCTs) where the degree of borrowing is determined based on similarity between RCT and external control patients to account for systematic differences (e.g., unmeasured confounders). The method represents a novel extension of the power prior where discounting weights are computed separately for each external control based on compatibility with the randomized control data. The discounting weights are determined using the predictive distribution for the external controls derived via the posterior distribution for time-to-event parameters estimated from the RCT. This method is applied using a proportional hazards regression model with piecewise constant baseline hazard. A simulation study and a real-data example are presented based on a completed trial in non-small cell lung cancer. It is shown that the case weighted power prior provides robust inference under various forms of incompatibility between the external controls and RCT population.

摘要

我们开发了一种混合分析方法,该方法使用外部对照来增强随机对照试验(RCT)中的内部对照臂,其中借用程度基于 RCT 和外部对照患者之间的相似性来确定,以考虑系统差异(例如,未测量的混杂因素)。该方法代表了功效先验的一种新扩展,其中为每个外部对照单独计算折扣权重,这是基于与随机对照数据的兼容性。折扣权重是通过从 RCT 中估计的时间事件参数的后验分布推导出的外部控制的预测分布来确定的。该方法应用于具有分段常数基线风险的比例风险回归模型。基于非小细胞肺癌完成的试验,提出了一项模拟研究和一个真实数据示例。结果表明,在外部对照和 RCT 人群之间存在各种形式的不兼容的情况下,病例加权功效先验提供了稳健的推断。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7ea5/10968526/077f7b9b2f6e/ujae019fig1.jpg

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