arXivDaily arXiv每日学术速递 周一至周五更新
arXiv 2004.14824stat.MEmath.STstat.TH
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竞赛项目中可分离效应的广义解释和识别

Generalized interpretation and identification of separable effects in competing event settings

Mats J. Stensrud, Miguel A. Hernán, Eric J. Tchetgen Tchetgen, James M. Robins, Vanessa Didelez, Jessica G. Young

摘要:

在竞争事件背景下,原因特异性累积发生率的反事实对比量化了治疗对关注事件的总体因果效应。然而,治疗对比赛项目的影响可能间接地促成了这一总效应,使其解释复杂化。我们之前提出了可分离效应(Stensrud et al,2019),以定义治疗对关注事件的直接和间接效应。该定义的前提是将治疗分解为两个成分,沿着两个独立的因果路径起作用,一个完全在竞争事件之外,另一个完全通过竞争事件。与以前的直接和间接效应定义不同,在对治疗成分进行单独干预的研究中,可以对可分离效应进行实证审查。在这里,我们扩展和推广的概念,可分离的影响,在几个方面,允许解释,识别和估计下相当弱的假设。我们提出并讨论了一个定义的可分离的效果,适用于一般的时变结构,其中的可分离的效果仍然可以有意义地解释,即使它们不能被视为直接和间接的影响。我们进一步推导出在尚未提供分解治疗的观察性研究中识别可分离效应的较弱条件;特别是,这些条件允许感兴趣事件、竞争事件和失访的常见原因随时间变化。对于这些一般设置,我们提出了半参数加权估计,是直接实现。作为一个例子,我们应用估计量来研究强化血压治疗对急性肾损伤的可分离影响,使用随机临床试验的数据。

英文摘要:

In competing event settings, a counterfactual contrast of cause-specific cumulative incidences quantifies the total causal effect of a treatment on the event of interest. However, effects of treatment on the competing event may indirectly contribute to this total effect, complicating its interpretation. We previously proposed the separable effects (Stensrud et al, 2019) to define direct and indirect effects of the treatment on the event of interest. This definition presupposes a treatment decomposition into two components acting along two separate causal pathways, one exclusively outside of the competing event and the other exclusively through it. Unlike previous definitions of direct and indirect effects, the separable effects can be subject to empirical scrutiny in a study where separate interventions on the treatment components are available. Here we extend and generalize the notion of the separable effects in several ways, allowing for interpretation, identification and estimation under considerably weaker assumptions. We propose and discuss a definition of separable effects that is applicable to general time-varying structures, where the separable effects can still be meaningfully interpreted, even when they cannot be regarded as direct and indirect effects. We further derive weaker conditions for identification of separable effects in observational studies where decomposed treatments are not yet available; in particular, these conditions allow for time-varying common causes of the event of interest, the competing events and loss to follow-up. For these general settings, we propose semi-parametric weighted estimators that are straightforward to implement. As an illustration, we apply the estimators to study the separable effects of intensive blood pressure therapy on acute kidney injury, using data from a randomized clinical trial.

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