Biostatistics, Massachusetts General Hospital, Boston, Massachusetts, USA.
Department of Medicine, Harvard Medical School, Boston, Massachusetts, USA.
Stat Med. 2024 Mar 30;43(7):1458-1474. doi: 10.1002/sim.10027. Epub 2024 Feb 5.
Generalized estimating equations (GEEs) provide a useful framework for estimating marginal regression parameters based on data from cluster randomized trials (CRTs), but they can result in inaccurate parameter estimates when some outcomes are informatively missing. Existing techniques to handle missing outcomes in CRTs rely on correct specification of a propensity score model, a covariate-conditional mean outcome model, or require at least one of these two models to be correct, which can be challenging in practice. In this article, we develop new weighted GEEs to simultaneously estimate the marginal mean, scale, and correlation parameters in CRTs with missing outcomes, allowing for multiple propensity score models and multiple covariate-conditional mean models to be specified. The resulting estimators are consistent provided that any one of these models is correct. An iterative algorithm is provided for implementing this more robust estimator and practical considerations for specifying multiple models are discussed. We evaluate the performance of the proposed method through Monte Carlo simulations and apply the proposed multiply robust estimator to analyze the Botswana Combination Prevention Project, a large HIV prevention CRT designed to evaluate whether a combination of HIV-prevention measures can reduce HIV incidence.
广义估计方程(GEE)为基于群组随机试验(CRT)数据估计边缘回归参数提供了一个有用的框架,但当某些结局存在信息缺失时,它们可能会导致不准确的参数估计。现有的处理 CRT 中缺失结局的技术依赖于正确指定倾向评分模型、协变量条件均值结局模型,或者至少要求这两个模型中的一个正确,这在实践中可能具有挑战性。在本文中,我们开发了新的加权 GEE,用于同时估计具有缺失结局的 CRT 中的边缘均值、比例和相关参数,允许指定多个倾向评分模型和多个协变量条件均值模型。只要其中任何一个模型正确,得到的估计量就是一致的。我们提供了一个迭代算法来实现这个更稳健的估计器,并讨论了指定多个模型的实际考虑因素。我们通过蒙特卡罗模拟评估了所提出方法的性能,并将所提出的多重稳健估计器应用于分析博茨瓦纳联合预防项目,这是一个大型的 HIV 预防 CRT,旨在评估 HIV 预防措施的组合是否可以降低 HIV 发病率。