随机试验中竞争风险数据的因果效应估计:调整协变量以获得效率。

IF 1.1 4区 数学 Q2 STATISTICS & PROBABILITY
Journal of Applied Statistics Pub Date : 2025-01-24 eCollection Date: 2025-01-01 DOI:10.1080/02664763.2025.2455626
Youngjoo Cho, Cheng Zheng, Lihong Qi, Ross L Prentice, Mei-Jie Zhang
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引用次数: 0

摘要

双盲随机试验被认为是估计平均因果效应(ACE)的黄金标准。不调整任何协变量的朴素估计量是一致的。然而,纳入强预测结果的协变量可以减少治疗组和对照组之间协变量分布不平衡的问题,并可以提高效率。最近的研究表明,由于随机化,对于线性回归,即使假设非参数模型对协变量的影响,回归系数在风险一致性下的估计器(例如随机森林)也可以保持收敛速度。此外,与未经调整的估计器相比,这种调整后的估计器总是会导致效率提高。本文将这一结果推广到竞争风险数据集,并证明在相似的假设条件下,基于增广逆概率滤波加权(AIPCW)的调整估计量具有相同的收敛速度和效率增益。进行了大量的模拟,以显示有限样本设置下的效率增益。为了说明我们提出的方法,我们将其应用于妇女健康倡议(WHI)饮食调整试验,研究低脂饮食对心血管疾病(CVD)相关死亡率的影响。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Causal effect estimation for competing risk data in randomized trial: adjusting covariates to gain efficiency.

The double-blinded randomized trial is considered the gold standard to estimate the average causal effect (ACE). The naive estimator without adjusting any covariate is consistent. However, incorporating the covariates that are strong predictors of the outcome could reduce the issue of unbalanced covariate distribution between the treated and controlled groups and can improve efficiency. Recent work has shown that thanks to randomization, for linear regression, an estimator under risk consistency (e.g. Random Forest) for the regression coefficients could maintain the convergence rate even when a nonparametric model is assumed for the effect of covariates. Also, such an adjusted estimator will always lead to efficiency gain compared to the naive unadjusted estimator. In this paper, we extend this result to the competing risk data setting and show that under similar assumptions, the augmented inverse probability censoring weighting (AIPCW) based adjusted estimator has the same convergence rate and efficiency gain. Extensive simulations were performed to show the efficiency gain in the finite sample setting. To illustrate our proposed method, we apply it to the Women's Health Initiative (WHI) dietary modification trial studying the effect of a low-fat diet on cardiovascular disease (CVD) related mortality among those who have prior CVD.

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来源期刊
Journal of Applied Statistics
Journal of Applied Statistics 数学-统计学与概率论
CiteScore
3.40
自引率
0.00%
发文量
126
审稿时长
6 months
期刊介绍: Journal of Applied Statistics provides a forum for communication between both applied statisticians and users of applied statistical techniques across a wide range of disciplines. These areas include business, computing, economics, ecology, education, management, medicine, operational research and sociology, but papers from other areas are also considered. The editorial policy is to publish rigorous but clear and accessible papers on applied techniques. Purely theoretical papers are avoided but those on theoretical developments which clearly demonstrate significant applied potential are welcomed. Each paper is submitted to at least two independent referees.
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