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Improve efficiency and reduce bias of Cox regression models for two-stage randomization designs using auxiliary covariates

机译:使用辅助协变量提高两阶段随机化设计的效率和减少COX回归模型的偏差

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摘要

Two-stage randomization designs are broadly accepted and becoming increasingly popular in clinical trials for cancer and other chronic diseases to assess and compare the effects of different treatment policies. In this paper, we propose an inferential method to estimate the treatment effects in two-stage randomization designs, which can improve the efficiency and reduce bias in the presence of chance imbalance of a robust covariate-adjustment without additional assumptions required by Lokhnygina and Helterbrand (Biometrics, 63:422-428)'s inverse probability weighting (IPW) method. The proposed method is evaluated and compared with the IPW method using simulations and an application to data from an oncology clinical trial. Given the predictive power of baseline covariates collected in this real data, our proposed method obtains 17-38% gains in efficiency compared with the IPW method in terms of overall survival outcome. Copyright (C) 2017 John Wiley & Sons, Ltd.
机译:两阶段随机化设计被广泛接受,在癌症和其他慢性疾病的临床试验中越来越受欢迎,以评估和比较不同治疗政策的影响。 在本文中,我们提出了一种推论方法来估算两阶段随机化设计中的治疗效果,这可以提高效率和减少稳健的协变量调节的效率,而没有Lokhnygina和Helterbrand所需的额外假设( Biometrics,63:422-428)的反概率加权(IPW)方法。 评估所提出的方法,并将其使用模拟的IPW方法与来自肿瘤临床试验的数据的应用程序进行比较。 鉴于在该实际数据收集的基线协变量的预测力,我们所提出的方法在整体存活结果方面与IPW方法相比,效率获得17-38%。 版权所有(C)2017 John Wiley&Sons,Ltd。

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