title: Identification of subgroups with differential treatment effects for longitudinal and multiresponse variables.
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. Copyright © 2016 John Wiley & Sons, Ltd.
authors.affiliation: Department of Statistics, University of Wisconsin, Madison, 53706, WI, U.S.A.. loh@stat.wisc.edu.
authors.forename: Wei-Yin
authors.initials: WY
authors.lastname: Loh
authors.affiliation: Eli Lilly Company, Indianapolis, 46285, IN, U.S.A.
authors.forename: Haoda
authors.initials: H
authors.lastname: Fu
authors.affiliation: Eli Lilly Company, Indianapolis, 46285, IN, U.S.A.
authors.forename: Michael
authors.initials: M
authors.lastname: Man
authors.affiliation: School of Nursing, Indiana University, Indianapolis, 46202, IN, U.S.A.
authors.forename: Victoria
authors.initials: V
authors.lastname: Champion
authors.affiliation: Department of Biostatistics & Medical Informatics, University of Wisconsin, Madison, 53706, WI, U.S.A.
authors.forename: Menggang
authors.initials: M
authors.lastname: Yu
mesh_terms: D000465:Algorithms; D003627:Data Interpretation, Statistical; D006801:Humans; D008137:Longitudinal Studies; D016032:Randomized Controlled Trials as Topic; D016896:Treatment Outcome
keywords: bootstrap; precision medicine; randomized trial; regression tree; unbiased