利用时间到事件数据进行混合控制分析的案例加权幂先验。

IF 1.4 4区 数学 Q3 BIOLOGY
Biometrics Pub Date : 2024-03-27 DOI:10.1093/biomtc/ujae019
Evan Kwiatkowski, Jiawen Zhu, Xiao Li, Herbert Pang, Grazyna Lieberman, Matthew A Psioda
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引用次数: 0

摘要

我们开发了一种混合分析方法,利用外部对照来增强随机对照试验(RCT)中的内部对照臂,根据 RCT 和外部对照患者的相似性来确定借用程度,以考虑系统性差异(如未测量的混杂因素)。该方法是对功率先验的新扩展,根据与随机对照数据的兼容性,分别计算每个外部对照的贴现权重。贴现权重是利用外部对照的预测分布确定的,预测分布是通过 RCT 估计的时间到事件参数的后验分布得出的。该方法使用的是具有片断恒定基线危害的比例危害回归模型。本文以一项已完成的非小细胞肺癌试验为基础,介绍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 群体之间存在各种形式的不相容性的情况下,病例加权功率先验可提供稳健的推断。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Case weighted power priors for hybrid control analyses with time-to-event data.

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.

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来源期刊
Biometrics
Biometrics 生物-生物学
CiteScore
2.70
自引率
5.30%
发文量
178
审稿时长
4-8 weeks
期刊介绍: The International Biometric Society is an international society promoting the development and application of statistical and mathematical theory and methods in the biosciences, including agriculture, biomedical science and public health, ecology, environmental sciences, forestry, and allied disciplines. The Society welcomes as members statisticians, mathematicians, biological scientists, and others devoted to interdisciplinary efforts in advancing the collection and interpretation of information in the biosciences. The Society sponsors the biennial International Biometric Conference, held in sites throughout the world; through its National Groups and Regions, it also Society sponsors regional and local meetings.
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