Systematically missing data in causally interpretable meta-analysis.

IF 1.8 3区 数学 Q3 MATHEMATICAL & COMPUTATIONAL BIOLOGY Biostatistics Pub Date : 2024-04-15 DOI:10.1093/biostatistics/kxad006
Jon A Steingrimsson, David H Barker, Ruofan Bie, Issa J Dahabreh
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Abstract

Causally interpretable meta-analysis combines information from a collection of randomized controlled trials to estimate treatment effects in a target population in which experimentation may not be possible but from which covariate information can be obtained. In such analyses, a key practical challenge is the presence of systematically missing data when some trials have collected data on one or more baseline covariates, but other trials have not, such that the covariate information is missing for all participants in the latter. In this article, we provide identification results for potential (counterfactual) outcome means and average treatment effects in the target population when covariate data are systematically missing from some of the trials in the meta-analysis. We propose three estimators for the average treatment effect in the target population, examine their asymptotic properties, and show that they have good finite-sample performance in simulation studies. We use the estimators to analyze data from two large lung cancer screening trials and target population data from the National Health and Nutrition Examination Survey (NHANES). To accommodate the complex survey design of the NHANES, we modify the methods to incorporate survey sampling weights and allow for clustering.

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可解释因果关系的荟萃分析中的系统缺失数据。
可解释因果关系的荟萃分析结合了一系列随机对照试验的信息,以估算目标人群的治疗效果,在目标人群中可能无法进行试验,但可以从中获得协变量信息。在此类分析中,一个关键的实际挑战是系统性数据缺失的存在,即某些试验收集了一个或多个基线协变量的数据,而其他试验却没有收集,从而导致后者所有参与者的协变量信息缺失。在本文中,我们将提供在荟萃分析中部分试验系统性缺失协变量数据时,目标人群中潜在(反事实)结果均值和平均治疗效果的识别结果。我们提出了目标人群平均治疗效果的三个估计值,考察了它们的渐近特性,并在模拟研究中证明它们具有良好的有限样本性能。我们使用这些估计值分析了两项大型肺癌筛查试验的数据以及美国国家健康与营养调查(NHANES)的目标人群数据。为了适应 NHANES 复杂的调查设计,我们对方法进行了修改,加入了调查抽样权重并允许聚类。
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来源期刊
Biostatistics
Biostatistics 生物-数学与计算生物学
CiteScore
5.10
自引率
4.80%
发文量
45
审稿时长
6-12 weeks
期刊介绍: Among the important scientific developments of the 20th century is the explosive growth in statistical reasoning and methods for application to studies of human health. Examples include developments in likelihood methods for inference, epidemiologic statistics, clinical trials, survival analysis, and statistical genetics. Substantive problems in public health and biomedical research have fueled the development of statistical methods, which in turn have improved our ability to draw valid inferences from data. The objective of Biostatistics is to advance statistical science and its application to problems of human health and disease, with the ultimate goal of advancing the public''s health.
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