Sample Size Requirements to Test Subgroup-Specific Treatment Effects in Cluster-Randomized Trials.

IF 3 2区 医学 Q2 PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH
Prevention Science Pub Date : 2024-07-01 Epub Date: 2023-10-10 DOI:10.1007/s11121-023-01590-6
Xueqi Wang, Keith S Goldfeld, Monica Taljaard, Fan Li
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Abstract

Cluster-randomized trials (CRTs) often allocate intact clusters of participants to treatment or control conditions and are increasingly used to evaluate healthcare delivery interventions. While previous studies have developed sample size methods for testing confirmatory hypotheses of treatment effect heterogeneity in CRTs (i.e., targeting the difference between subgroup-specific treatment effects), sample size methods for testing the subgroup-specific treatment effects themselves have not received adequate attention-despite a rising interest in health equity considerations in CRTs. In this article, we develop formal methods for sample size and power analyses for testing subgroup-specific treatment effects in parallel-arm CRTs with a continuous outcome and a binary subgroup variable. We point out that the variances of the subgroup-specific treatment effect estimators and their covariance are given by weighted averages of the variance of the overall average treatment effect estimator and the variance of the heterogeneous treatment effect estimator. This analytical insight facilitates an explicit characterization of the requirements for both the omnibus test and the intersection-union test to achieve the desired level of power. Generalizations to allow for subgroup-specific variance structures are also discussed. We report on a simulation study to validate the proposed sample size methods and demonstrate that the empirical power corresponds well with the predicted power for both tests. The design and setting of the Umea Dementia and Exercise (UMDEX) CRT in older adults are used to illustrate our sample size methods.

Abstract Image

在集群随机试验中测试亚组特异性治疗效果的样本量要求。
集群随机试验(CRT)通常将完整的参与者集群分配给治疗或对照条件,并越来越多地用于评估医疗保健干预措施。虽然先前的研究已经开发了样本量方法来测试CRT中治疗效果异质性的验证性假设(即针对亚组特异性治疗效果之间的差异),尽管人们对CRT中的健康公平考虑越来越感兴趣,但用于测试亚组特异性治疗效果的样本量方法本身并没有得到足够的关注。在这篇文章中,我们开发了样本量和功率分析的正式方法,用于测试具有连续结果和二元亚组变量的平行臂CRT中的亚组特异性治疗效果。我们指出,亚群特定治疗效果估计量的方差及其协方差是由整体平均治疗效果估计数的方差和异质治疗效果估计值的方差的加权平均值给出的。这种分析见解有助于明确描述综合测试和交叉并集测试的要求,以达到所需的功率水平。还讨论了允许子群特定方差结构的推广。我们报告了一项模拟研究,以验证所提出的样本量方法,并证明经验功率与两种测试的预测功率非常一致。老年痴呆症和运动(UMDEX)CRT的设计和设置用于说明我们的样本量方法。
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来源期刊
Prevention Science
Prevention Science PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH-
CiteScore
6.50
自引率
11.40%
发文量
128
期刊介绍: Prevention Science is the official publication of the Society for Prevention Research. The Journal serves as an interdisciplinary forum designed to disseminate new developments in the theory, research and practice of prevention. Prevention sciences encompassing etiology, epidemiology and intervention are represented through peer-reviewed original research articles on a variety of health and social problems, including but not limited to substance abuse, mental health, HIV/AIDS, violence, accidents, teenage pregnancy, suicide, delinquency, STD''s, obesity, diet/nutrition, exercise, and chronic illness. The journal also publishes literature reviews, theoretical articles, meta-analyses, systematic reviews, brief reports, replication studies, and papers concerning new developments in methodology.
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