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Change-plane analysis for subgroup detection with a continuous treatment.


ABSTRACT: Detecting and characterizing subgroups with differential effects of a binary treatment has been widely studied and led to improvements in patient outcomes and population risk management. Under the setting of a continuous treatment, however, such investigations remain scarce. We propose a semiparametric change-plane model and consequently a doubly robust test statistic for assessing the existence of two subgroups with differential treatment effects under a continuous treatment. The proposed testing procedure is valid when either the baseline function for the covariate effects or the generalized propensity score function for the continuous treatment is correctly specified. The asymptotic distributions of the test statistic under the null and local alternative hypotheses are established. When the null hypothesis of no subgroup is rejected, the change-plane parameters that define the subgroups can be estimated. This paper provides a unified framework of the change-plane method to handle various types of outcomes, including the exponential family of distributions and time-to-event outcomes. Additional extensions with nonparametric estimation approaches are also provided. We evaluate the performance of our proposed methods through extensive simulation studies under various scenarios. An application to the Health Effects of Arsenic Longitudinal Study with a continuous environmental exposure of arsenic is presented.

SUBMITTER: Jin P 

PROVIDER: S-EPMC10030385 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature

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Change-plane analysis for subgroup detection with a continuous treatment.

Jin Peng P   Lu Wenbin W   Chen Yu Y   Liu Mengling M  

Biometrics 20221024 3


Detecting and characterizing subgroups with differential effects of a binary treatment has been widely studied and led to improvements in patient outcomes and population risk management. Under the setting of a continuous treatment, however, such investigations remain scarce. We propose a semiparametric change-plane model and consequently a doubly robust test statistic for assessing the existence of two subgroups with differential treatment effects under a continuous treatment. The proposed testi  ...[more]

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