Eli Lilly and Company, Indianapolis, IN 46204, USA.
Department of Statistics, North Carolina State University, SAS Hall, 2311 Stinson Dr, Raleigh, NC 27607, USA.
Biostatistics. 2024 Oct 1;25(4):933-946. doi: 10.1093/biostatistics/kxae002.
Clinicians and patients must make treatment decisions at a series of key decision points throughout disease progression. A dynamic treatment regime is a set of sequential decision rules that return treatment decisions based on accumulating patient information, like that commonly found in electronic medical record (EMR) data. When applied to a patient population, an optimal treatment regime leads to the most favorable outcome on average. Identifying optimal treatment regimes that maximize residual life is especially desirable for patients with life-threatening diseases such as sepsis, a complex medical condition that involves severe infections with organ dysfunction. We introduce the residual life value estimator (ReLiVE), an estimator for the expected value of cumulative restricted residual life under a fixed treatment regime. Building on ReLiVE, we present a method for estimating an optimal treatment regime that maximizes expected cumulative restricted residual life. Our proposed method, ReLiVE-Q, conducts estimation via the backward induction algorithm Q-learning. We illustrate the utility of ReLiVE-Q in simulation studies, and we apply ReLiVE-Q to estimate an optimal treatment regime for septic patients in the intensive care unit using EMR data from the Multiparameter Intelligent Monitoring Intensive Care database. Ultimately, we demonstrate that ReLiVE-Q leverages accumulating patient information to estimate personalized treatment regimes that optimize a clinically meaningful function of residual life.
临床医生和患者必须在疾病进展的一系列关键决策点做出治疗决策。动态治疗方案是一组基于累积患者信息的序贯决策规则,例如在电子病历 (EMR) 数据中常见的信息。当应用于患者群体时,最优治疗方案可平均实现最佳结果。对于脓毒症等危及生命的疾病患者,确定可最大限度延长剩余寿命的最优治疗方案尤为重要,脓毒症是一种复杂的医疗状况,涉及严重感染和器官功能障碍。我们引入了剩余寿命值估计器 (ReLiVE),这是一种用于在固定治疗方案下累积受限剩余寿命的期望值的估计器。基于 ReLiVE,我们提出了一种用于估计可最大化预期累积受限剩余寿命的最优治疗方案的方法。我们提出的方法 ReLiVE-Q 通过反向归纳算法 Q-学习进行估计。我们通过模拟研究说明了 ReLiVE-Q 的实用性,并在 Multiparameter Intelligent Monitoring Intensive Care 数据库的 EMR 数据中应用 ReLiVE-Q 来估计重症监护病房中脓毒症患者的最优治疗方案。最终,我们证明了 ReLiVE-Q 利用累积的患者信息来估计个性化的治疗方案,从而优化了剩余寿命的临床有意义的功能。