Survival causal rule ensemble method considering the main effect for estimating heterogeneous treatment effects

Stat Med. 2024 Nov 30;43(27):5234-5271. doi: 10.1002/sim.10180.

Abstract

With an increasing focus on precision medicine in medical research, numerous studies have been conducted in recent years to clarify the relationship between treatment effects and patient characteristics. The treatment effects for patients with different characteristics are always heterogeneous, and therefore, various heterogeneous treatment effect machine learning estimation methods have been proposed owing to their flexibility and high estimation accuracy. However, most machine learning methods rely on black-box models, preventing direct interpretation of the relationship between patient characteristics and treatment effects. Moreover, most of these studies have focused on continuous or binary outcomes, although survival outcomes are also important in medical research. To address these challenges, we propose a heterogeneous treatment effect estimation method for survival data based on RuleFit, an interpretable machine learning method. Numerical simulation results confirmed that the prediction performance of the proposed method was comparable to that of existing methods. We also applied a dataset from an HIV study, the AIDS Clinical Trials Group Protocol 175 dataset, to illustrate the interpretability of the proposed method using real data. Consequently, the proposed survival causal rule ensemble method provides an interpretable model with sufficient estimation accuracy.

Keywords: heterogeneous treatment effect; interpretability; randomized control trial; rule ensemble; survival analysis.

MeSH terms

  • Computer Simulation*
  • HIV Infections / drug therapy
  • HIV Infections / mortality
  • Humans
  • Machine Learning*
  • Models, Statistical
  • Precision Medicine / methods
  • Survival Analysis
  • Treatment Effect Heterogeneity
  • Treatment Outcome