Identification of subgroups with differential treatment effects for longitudinal and multiresponse variables

Wei Yin Loh, Haoda Fu, Michael Man, Victoria Champion, Menggang Yu

Research output: Contribution to journalArticle

11 Scopus citations

Abstract

We describe and evaluate a regression tree algorithm for finding subgroups with differential treatments effects in randomized trials with multivariate outcomes. The data may contain missing values in the outcomes and covariates, and the treatment variable is not limited to two levels. Simulation results show that the regression tree models have unbiased variable selection and the estimates of subgroup treatment effects are approximately unbiased. A bootstrap calibration technique is proposed for constructing confidence intervals for the treatment effects. The method is illustrated with data from a longitudinal study comparing two diabetes drugs and a mammography screening trial comparing two treatments and a control.

Original languageEnglish (US)
Pages (from-to)4837-4855
Number of pages19
JournalStatistics in Medicine
Volume35
Issue number26
DOIs
StatePublished - Nov 20 2016

Keywords

  • bootstrap
  • precision medicine
  • randomized trial
  • regression tree
  • unbiased

ASJC Scopus subject areas

  • Epidemiology
  • Statistics and Probability

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