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

📅 2025-07-26
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🤖 AI Summary
Alcohol Use Disorder (AUD) treatment exhibits substantial inter-individual heterogeneity, and drinking outcomes follow a zero-one-inflated semi-continuous distribution—posing significant challenges for personalized treatment effect prediction and decision-making. To address this, we propose HOBZ-BART: a novel hierarchical Bayesian model that decomposes outcomes into three mutually exclusive components—zero, positive continuous, and one—via a tripartite hurdle structure. It jointly models nonlinear main effects, covariate interactions, and personalized treatment effects (PITE) by integrating Beta-likelihood approximation within a shared Bayesian Additive Regression Trees (BART) framework. The model supports full Bayesian posterior inference, ensuring both high predictive accuracy and intrinsic interpretability. Evaluated on the MATCH clinical trial dataset, HOBZ-BART significantly outperforms benchmark models—including zero-one-inflated beta (ZOIB)—in estimating heterogeneous treatment effects across psychological interventions. It delivers actionable, interpretable insights for tailoring AUD interventions to individual patients.

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📝 Abstract
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.
Problem

Research questions and friction points this paper is trying to address.

Modeling zero and one inflated data for AUD treatment outcomes
Estimating predictive individual treatment effects (PITEs) accurately
Comparing CBT, MET, and TSF therapies for personalized insights
Innovation

Methods, ideas, or system contributions that make the work stand out.

Bayesian Additive Regression Trees for semicontinuous data
Sequential hurdle structure for clinical interpretability
Scalable Beta-likelihood approximation for efficient computation
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