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A model-based multithreshold method for subgroup identification.

Jingli WangJialiang LiYaguang LiWeng Kee Wong
Published in: Statistics in medicine (2019)
Thresholding variable plays a crucial role in subgroup identification for personalized medicine. Most existing partitioning methods split the sample based on one predictor variable. In this paper, we consider setting the splitting rule from a combination of multivariate predictors, such as the latent factors, principle components, and weighted sum of predictors. Such a subgrouping method may lead to more meaningful partitioning of the population than using a single variable. In addition, our method is based on a change point regression model and thus yields straight forward model-based prediction results. After choosing a particular thresholding variable form, we apply a two-stage multiple change point detection method to determine the subgroups and estimate the regression parameters. We show that our approach can produce two or more subgroups from the multiple change points and identify the true grouping with high probability. In addition, our estimation results enjoy oracle properties. We design a simulation study to compare performances of our proposed and existing methods and apply them to analyze data sets from a Scleroderma trial and a breast cancer study.
Keyphrases
  • phase iii
  • clinical trial
  • systemic sclerosis
  • magnetic resonance
  • study protocol
  • young adults
  • machine learning
  • open label
  • interstitial lung disease
  • data analysis
  • deep learning
  • double blind