Optimizing personalized treatments for targeted patient populations across multiple domains

Learning individualized treatment rules (ITRs) for a target patient population with mental disorders is confronted with many challenges. First, the target population may be different from the training population that provided data for learning ITRs. Ignoring differences between the training patient data and the target population can result in sub-optimal treatment strategies for the target population. Second, for mental disorders, a patient's underlying mental state is not observed but can be inferred from measures of high-dimensional combinations of symptomatology. Treatment mechanisms are unknown and can be complex, and thus treatment effect moderation can take complicated forms. To address these challenges, we propose a novel method that connects measurement models, efficient weighting schemes, and flexible neural network architecture through latent variables to tailor treatments for a target population. Patients' underlying mental states are represented by a compact set of latent state variables while preserving interpretability. Weighting schemes are designed based on lower-dimensional latent variables to efficiently balance population differences so that biases in learning the latent structure and treatment effects are mitigated. Extensive simulation studies demonstrated consistent superiority of the proposed method and the weighting approach. Applications to two real-world studies of patients with major depressive disorder have shown a broad utility of the proposed method in improving treatment outcomes in the target population.

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Publication Details

Journal
The International Journal of Biostatistics
Published
2024-09-26
DOI
https://doi.org/10.1515/ijb-2024-0068
Citations
1
Primary Topic
Advanced Causal Inference Techniques
Type
article
Field-Weighted Citation Impact
0.71

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article

Optimizing personalized treatments for targeted patient populations across multiple domains

Donglin Zeng, Yuanjia Wang, Yuan Chen
1 citations
The International Journal of Biostatistics
Advanced Causal Inference Techniques
0.71
article

Optimizing personalized treatments for targeted patient populations across multiple domains

Donglin Zeng, Yuanjia Wang, Yuan Chen
article en
1 citations

Abstract

Learning individualized treatment rules (ITRs) for a target patient population with mental disorders is confronted with many challenges. First, the target population may be different from the training population that provided data for learning ITRs. Ignoring differences between the training patient data and the target population can result in sub-optimal treatment strategies for the target population. Second, for mental disorders, a patient's underlying mental state is not observed but can be inferred from measures of high-dimensional combinations of symptomatology. Treatment mechanisms are unknown and can be complex, and thus treatment effect moderation can take complicated forms. To address these challenges, we propose a novel method that connects measurement models, efficient weighting schemes, and flexible neural network architecture through latent variables to tailor treatments for a target population. Patients' underlying mental states are represented by a compact set of latent state variables while preserving interpretability. Weighting schemes are designed based on lower-dimensional latent variables to efficiently balance population differences so that biases in learning the latent structure and treatment effects are mitigated. Extensive simulation studies demonstrated consistent superiority of the proposed method and the weighting approach. Applications to two real-world studies of patients with major depressive disorder have shown a broad utility of the proposed method in improving treatment outcomes in the target population.

The International Journal of BiostatisticsVol. 20(2)
Memorial Sloan Kettering Cancer Center (US), University of Michigan (US), Columbia University (US)
Division of Cancer Prevention, National Cancer Institute, National Institutes of Health, National Institute of Mental Health, National Institute of General Medical Sciences, National Institute of Neurological Disorders and Stroke
Openalex Percentile: Top 26%
Advanced Causal Inference Techniques
0.71
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