论文标题
反事实表型,审查的事件时间
Counterfactual Phenotyping with Censored Time-to-Events
论文作者
论文摘要
现实世界中临床干预措施的治疗效果的估计涉及处理诸如死亡时间,重新住院或可能受到检查的复合事件之类的连续结果。在这种情况下,反事实推理需要将影响基线存活率的混杂生理特征的影响与所评估的干预措施的影响产生影响。在本文中,我们提出一种潜在变量方法来模拟异质治疗效应,方法是提出一个人可以属于具有不同响应特征的潜在簇之一。我们表明,这种潜在结构可以介导基本的生存率,并有助于确定干预的影响。我们证明了我们的方法基于对个体的治疗反应发现可起作用的表型的能力,该反应最初是针对评估适当治疗以减少心血管风险的多个大型随机临床试验的能力。
Estimation of treatment efficacy of real-world clinical interventions involves working with continuous outcomes such as time-to-death, re-hospitalization, or a composite event that may be subject to censoring. Counterfactual reasoning in such scenarios requires decoupling the effects of confounding physiological characteristics that affect baseline survival rates from the effects of the interventions being assessed. In this paper, we present a latent variable approach to model heterogeneous treatment effects by proposing that an individual can belong to one of latent clusters with distinct response characteristics. We show that this latent structure can mediate the base survival rates and helps determine the effects of an intervention. We demonstrate the ability of our approach to discover actionable phenotypes of individuals based on their treatment response on multiple large randomized clinical trials originally conducted to assess appropriate treatments to reduce cardiovascular risk.