Estimating heterogeneous survival treatment effect in observational data using machine learning

Liangyuan Hu, Jiayi Ji, Fan Li

Research output: Contribution to journalArticlepeer-review

34 Scopus citations

Abstract

Methods for estimating heterogeneous treatment effect in observational data have largely focused on continuous or binary outcomes, and have been relatively less vetted with survival outcomes. Using flexible machine learning methods in the counterfactual framework is a promising approach to address challenges due to complex individual characteristics, to which treatments need to be tailored. To evaluate the operating characteristics of recent survival machine learning methods for the estimation of treatment effect heterogeneity and inform better practice, we carry out a comprehensive simulation study presenting a wide range of settings describing confounded heterogeneous survival treatment effects and varying degrees of covariate overlap. Our results suggest that the nonparametric Bayesian Additive Regression Trees within the framework of accelerated failure time model (AFT-BART-NP) consistently yields the best performance, in terms of bias, precision, and expected regret. Moreover, the credible interval estimators from AFT-BART-NP provide close to nominal frequentist coverage for the individual survival treatment effect when the covariate overlap is at least moderate. Including a nonparametrically estimated propensity score as an additional fixed covariate in the AFT-BART-NP model formulation can further improve its efficiency and frequentist coverage. Finally, we demonstrate the application of flexible causal machine learning estimators through a comprehensive case study examining the heterogeneous survival effects of two radiotherapy approaches for localized high-risk prostate cancer.

Original languageEnglish
Pages (from-to)4691-4713
Number of pages23
JournalStatistics in Medicine
Volume40
Issue number21
DOIs
StatePublished - 20 Sep 2021

Keywords

  • Bayesian additive regression trees
  • causal inference
  • machine learning
  • observational studies
  • survival treatment effect heterogeneity

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