A Bayesian Additive Regression Trees Model for zero and one inflated data for Predicting Individual Treatment Effects in Alcohol Use Disorder Trials

Fuente: arXiv
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Main Authors: Solano, Pamela, Van Horn, M Lee, Walters, Kyle, Besendorfer, Philipp, Kuhlemeier, Alena, Martínez-Ramón, Manel, Jaki, Thomas
Format: Preprint
Published: 2025
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author Solano, Pamela
Van Horn, M Lee
Walters, Kyle
Besendorfer, Philipp
Kuhlemeier, Alena
Martínez-Ramón, Manel
Jaki, Thomas
author_facet Solano, Pamela
Van Horn, M Lee
Walters, Kyle
Besendorfer, Philipp
Kuhlemeier, Alena
Martínez-Ramón, Manel
Jaki, Thomas
contents Alcohol Use Disorder (AUD) treatment presents high individual-level heterogeneity, with outcomes ranging from complete abstinence to persistent heavy drinking. This variability-driven by complex behavioral, social, and environmental factors-poses major challenges for treatment evaluation and individualized decision-making. In particular, accurately modeling bounded semicontinuous outcomes and estimating predictive individual treatment effects (PITEs) remains methodologically demanding. For the pre-registered PITE analysis of Project MATCH, we developed HOBZ-BART, a novel Bayesian nonparametric model tailored for semicontinuous outcomes concentrated at clinically meaningful boundary values (0 and 1). The model decomposes the outcome into three components-abstinence, partial drinking, and persistent use-via a sequential hurdle structure, offering interpretability aligned with clinical reasoning. A shared Bayesian Additive Regression Tree (BART) ensemble captures nonlinear effects and covariate interactions across components, while a scalable Beta-likelihood approximation enables efficient, conjugate-friendly posterior computation. Through extensive simulations we demonstrate that HOBZ-BART outperforms traditional zero-one inflated Beta (ZOIB) model in predictive accuracy, computational efficiency, and PITE estimation. We then present the primary PITE analysis of the MATCH trial using HOBZ-BART which enables clinically meaningful comparisons of Cognitive Behavioral Therapy (CBT), Motivational Enhancement Therapy (MET), and Twelve Step Facilitation (TSF), offering personalized treatment insights. HOBZ-BART combines statistical rigor with clinical interpretability, addressing a critical need in addiction research for models that support individualized, data-driven care.
format Preprint
id arxiv_https___arxiv_org_abs_2507_19848
institution arXiv
publishDate 2025
record_format arxiv
spellingShingle A Bayesian Additive Regression Trees Model for zero and one inflated data for Predicting Individual Treatment Effects in Alcohol Use Disorder Trials
Solano, Pamela
Van Horn, M Lee
Walters, Kyle
Besendorfer, Philipp
Kuhlemeier, Alena
Martínez-Ramón, Manel
Jaki, Thomas
Applications
Alcohol Use Disorder (AUD) treatment presents high individual-level heterogeneity, with outcomes ranging from complete abstinence to persistent heavy drinking. This variability-driven by complex behavioral, social, and environmental factors-poses major challenges for treatment evaluation and individualized decision-making. In particular, accurately modeling bounded semicontinuous outcomes and estimating predictive individual treatment effects (PITEs) remains methodologically demanding. For the pre-registered PITE analysis of Project MATCH, we developed HOBZ-BART, a novel Bayesian nonparametric model tailored for semicontinuous outcomes concentrated at clinically meaningful boundary values (0 and 1). The model decomposes the outcome into three components-abstinence, partial drinking, and persistent use-via a sequential hurdle structure, offering interpretability aligned with clinical reasoning. A shared Bayesian Additive Regression Tree (BART) ensemble captures nonlinear effects and covariate interactions across components, while a scalable Beta-likelihood approximation enables efficient, conjugate-friendly posterior computation. Through extensive simulations we demonstrate that HOBZ-BART outperforms traditional zero-one inflated Beta (ZOIB) model in predictive accuracy, computational efficiency, and PITE estimation. We then present the primary PITE analysis of the MATCH trial using HOBZ-BART which enables clinically meaningful comparisons of Cognitive Behavioral Therapy (CBT), Motivational Enhancement Therapy (MET), and Twelve Step Facilitation (TSF), offering personalized treatment insights. HOBZ-BART combines statistical rigor with clinical interpretability, addressing a critical need in addiction research for models that support individualized, data-driven care.
title A Bayesian Additive Regression Trees Model for zero and one inflated data for Predicting Individual Treatment Effects in Alcohol Use Disorder Trials
topic Applications
url https://arxiv.org/abs/2507.19848