We focus on the problem of generalizing a causal effect estimated on a randomized controlled trial (RCT) to a target population described by a set of covariates from observational data. Available methods such as inverse propensity sampling weighting are not designed to handle missing values, which are however common in both data sources. In addition to coupling the assumptions for causal effect identifiability and for the missing values mechanism and to defining appropriate estimation strategies, one difficulty is to consider the specific structure of the data with two sources and treatment and outcome only available in the RCT. We propose three multiple imputation strategies to handle missing values when generalizing treatment effects, each handling the multi-source structure of the problem differently (separate imputation, joint imputation with fixed effect, joint imputation ignoring source information). As an alternative to multiple imputation, we also propose a direct estimation approach that treats incomplete covariates as semi-discrete variables. The multiple imputation strategies and the latter alternative rely on different sets of assumptions concerning the impact of missing values on identifiability. We discuss these assumptions and assess the methods through an extensive simulation study. This work is motivated by the analysis of a large registry of over 20,000 major trauma patients and an RCT studying the effect of tranexamic acid administration on mortality in major trauma patients admitted to ICU. The analysis illustrates how the missing values handling can impact the conclusion about the effect generalized from the RCT to the target population.
翻译:我们的重点是将随机控制试验(RCT)估计的因果关系扩大到一组观察数据共同变量所描述的目标人群。现有的方法,例如反偏向抽样权重,不是为了处理缺失值,尽管这两种数据来源都常见。除了将因果关系的可识别性和缺失值机制的假设结合起来,以及确定适当的估计战略,一个困难是考虑数据的具体结构,有两个来源和治疗及结果,只有RCT才有。我们提出三种多重估算战略,以便在普及治疗效果时处理缺失值,每种方法处理问题的多来源结构不同(分别估算、共同估算和固定效果,联合估算不考虑源信息)。除了将因果关系可识别性和缺失值机制的假设和缺失值机制的假设结合起来之外,我们还提出一种直接估算方法,将不完整的变量视为半分解变量。多重估算战略和后一种选择取决于关于缺失值对可识别性影响的不同假设。我们讨论这些假设,并通过大规模模拟分析对20世纪50年代患者的大规模创伤性影响进行评估。我们通过大规模模拟研究研究研究这些假设和评估了大规模创伤性病理病理的诊断方法。</s>