在结果缺失的随机临床试验中,基于经验似然法对平均治疗效果进行加权估计

Pub Date : 2024-07-19 DOI:10.4310/sii.2024.v17.n4.a7
Yuanyao Tan, Xialing Wen, Wei Liang, Ying Yan
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引用次数: 0

摘要

人们越来越关注在随机临床试验中以客观、高效的方式对治疗效果进行估计的协变量调整。本文提出了一种基于经验似然法的加权方法,用于提取随机临床试验中可能存在的结果缺失的协变量信息。我们采用多元回归模型分别描述了数据缺失机制和协方差-结果关系。我们证明了所提出的估计方法适用于治疗效果的客观推断。从理论上讲,我们证明了所提出的方法具有多重稳健性和半参数效率。我们还进行了模拟和真实数据研究,以便与其他现有方法进行比较。
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Empirical likelihood-based weighted estimation of average treatment effects in randomized clinical trials with missing outcomes
There has been growing attention on covariate adjustment for treatment effect estimation in an objective and efficient manner in randomized clinical trials. In this paper, we propose a weighting approach to extract covariate information based on the empirical likelihood method for the randomized clinical trials with possible missingness in the outcomes. Multiple regression models are imposed to delineate the missing data mechanism and the covariate-outcome relationship, respectively. We demonstrate that the proposed estimator is suitable for objective inference of treatment effects. Theoretically, we prove that the proposed approach is multiply robust and semiparametrically efficient. We conduct simulations and a real data study to make comparisons with other existing methods.
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