Estimation of optimal treatment regimes with electronic medical record data using the residual life value estimator

Biostatistics. 2024 Oct 1;25(4):933-946. doi: 10.1093/biostatistics/kxae002.

Abstract

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.

Keywords: MIMIC-III; Q-learning; context vector; dynamic treatment regime; electronic medical record; precision medicine; random forest; residual life; sepsis.

MeSH terms

  • Electronic Health Records*
  • Humans
  • Models, Statistical
  • Sepsis / therapy