Precision medicine: Subgroup identification in longitudinal trajectories.

Yishu Wei, Lei Liu, Xiaogang Su, Lihui Zhao, Hongmei Jiang
Author Information
  1. Yishu Wei: Department of statistics, Northwestern University, Evanston, IL, United States. ORCID
  2. Lei Liu: Division of biostatistics, Washington University, St. Louis, MO, United States. ORCID
  3. Xiaogang Su: Department of Mathematical Sciences, University of Texas at El Paso (UTEP), El Paso, TX, United States.
  4. Lihui Zhao: Northwestern University Feinberg School of Medicine, Chicago, IL, United States.
  5. Hongmei Jiang: Department of statistics, Northwestern University, Evanston, IL, United States.

Abstract

In clinical studies, the treatment effect may be heterogeneous among patients. It is of interest to identify subpopulations which benefit most from the treatment, regardless of the treatment's overall performance. In this study, we are interested in subgroup identification in longitudinal studies when nonlinear trajectory patterns are present. Under such a situation, evaluation of the treatment effect entails comparing longitudinal trajectories while subgroup identification requires a further evaluation of differential treatment effects among subgroups induced by moderators. To this end, we propose a tree-structured subgroup identification method, termed "interaction tree for longitudinal trajectories", which combines mixed effects models with regression splines to model the nonlinear progression patterns among repeated measures. Extensive simulation studies are conducted to evaluate its performance and an application to an alcohol addiction pharmacogenetic trial is presented.

Keywords

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Grants

  1. R01 HL136942/NHLBI NIH HHS
  2. UL1 TR002345/NCATS NIH HHS

MeSH Term

Algorithms
Computer Simulation
Humans
Longitudinal Studies
Pharmacogenetics
Precision Medicine

Word Cloud

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