Miles By C H, Shpitser I, Kanki P, Meloni S, Tchetgen E J Tchetgen
Department of Biostatistics, Columbia University Mailman School of Public Health, 722West 168th Street, NewYork, NewYork 10032, U.S.A.
Department of Computer Science, Johns Hopkins University, 160 Malone Hall, 3400 N. Charles Street, Baltimore, Maryland 21218, U.S.A.
Biometrika. 2020 Mar;107(1):159-172. doi: 10.1093/biomet/asz063. Epub 2019 Nov 23.
Path-specific effects constitute a broad class of mediated effects from an exposure to an outcome via one or more causal pathways along a set of intermediate variables. Most of the literature concerning estimation of mediated effects has focused on parametric models, with stringent assumptions regarding unmeasured confounding. We consider semiparametric inference of a path-specific effect when these assumptions are relaxed. In particular, we develop a suite of semiparametric estimators for the effect along a pathway through a mediator, but not through an exposure-induced confounder of that mediator. These estimators have different robustness properties, as each depends on different parts of the likelihood of the observed data. One estimator is locally semiparametric efficient and multiply robust. The latter property implies that machine learning can be used to estimate nuisance functions. We demonstrate these properties, as well as finite-sample properties of all the estimators, in a simulation study. We apply our method to an HIV study, in which we estimate the effect comparing two drug treatments on a patient's average log CD4 count mediated by the patient's level of adherence, but not by previous experience of toxicity, which is clearly affected by which treatment the patient is assigned to and may confound the effect of the patient's level of adherence on their virologic outcome.
路径特异性效应构成了一类广泛的介导效应,即从暴露经由一组中间变量沿着一条或多条因果路径到结局。大多数关于介导效应估计的文献都集中在参数模型上,对未测量的混杂因素有严格的假设。当这些假设放宽时,我们考虑路径特异性效应的半参数推断。具体而言,我们开发了一套半参数估计量,用于估计通过中介变量但不通过该中介变量的暴露诱导混杂因素的路径上的效应。这些估计量具有不同的稳健性,因为每个估计量都依赖于观测数据似然性的不同部分。一个估计量是局部半参数有效且多重稳健的。后一个性质意味着可以使用机器学习来估计干扰函数。我们在一项模拟研究中展示了这些性质以及所有估计量的有限样本性质。我们将我们的方法应用于一项HIV研究,在该研究中,我们估计比较两种药物治疗对患者平均log CD4计数的效应,该效应由患者的依从性水平介导,但不由先前的毒性经历介导,先前的毒性经历显然受患者所分配的治疗影响,并且可能混淆患者依从性水平对其病毒学结局的效应。