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用于同时学习最优个体化治疗规则和亚组的组合交互树。

Composite interaction tree for simultaneous learning of optimal individualized treatment rules and subgroups.

机构信息

Department of Biostatistics, Mailman School of Public Health, Columbia University, New York, New York.

Department of Psychiatry, Columbia University Medical Center, New York, New York.

出版信息

Stat Med. 2019 Jun 30;38(14):2632-2651. doi: 10.1002/sim.8105. Epub 2019 Mar 19.

DOI:10.1002/sim.8105
PMID:30891797
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8548070/
Abstract

Treatment response heterogeneity has long been observed in patients affected by chronic diseases. Administering an individualized treatment rule (ITR) offers an opportunity to tailor treatment strategies according to patient-specific characteristics. Overly complex machine learning methods for estimating ITRs may produce treatment rules that have higher benefit but lack transparency and interpretability. In clinical practices, it is desirable to derive a simple and interpretable ITR while maintaining certain optimality that leads to improved benefit in subgroups of patients, if not on the overall sample. In this work, we propose a tree-based robust learning method to estimate optimal piecewise linear ITRs and identify subgroups of patients with a large benefit. We achieve these goals by simultaneously identifying qualitative and quantitative interactions through a tree model, referred to as the composite interaction tree (CITree). We show that it has improved performance compared to existing methods on both overall sample and subgroups via extensive simulation studies. Lastly, we fit CITree to Research Evaluating the Value of Augmenting Medication with Psychotherapy trial for treating patients with major depressive disorders, where we identified both qualitative and quantitative interactions and subgroups of patients with a large benefit.

摘要

治疗反应的异质性在慢性疾病患者中早已得到观察。采用个体化治疗规则(ITR)可以根据患者的具体特征定制治疗策略。对于估计 ITR 的过于复杂的机器学习方法可能会产生具有更高收益但缺乏透明度和可解释性的治疗规则。在临床实践中,期望获得简单且可解释的 ITR,同时保持一定的最优性,从而为患者亚组带来改善的收益,如果不是对整个样本的话。在这项工作中,我们提出了一种基于树的稳健学习方法来估计最优分段线性 ITR,并识别具有较大收益的患者亚组。我们通过树模型(称为复合交互树(CITree))同时识别定性和定量交互,从而实现了这些目标。我们通过广泛的模拟研究表明,它在整体样本和亚组方面的性能均优于现有方法。最后,我们将 CITree 拟合到 Research Evaluating the Value of Augmenting Medication with Psychotherapy 试验中,用于治疗患有重度抑郁症的患者,我们在其中识别了定性和定量交互以及具有较大收益的患者亚组。

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本文引用的文献

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Augmented outcome-weighted learning for estimating optimal dynamic treatment regimens.增强型结果加权学习估计最优动态治疗方案。
Stat Med. 2018 Nov 20;37(26):3776-3788. doi: 10.1002/sim.7844. Epub 2018 Jun 5.
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Estimation and evaluation of linear individualized treatment rules to guarantee performance.线性个体化治疗规则的估计与评估以确保疗效。
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Qualitative interaction trees: a tool to identify qualitative treatment-subgroup interactions.定性交互树:一种用于识别定性治疗亚组交互作用的工具。
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Dysfunctional attitudes as a moderator of pharmacotherapy and psychotherapy for chronic depression.功能失调态度作为慢性抑郁症药物治疗和心理治疗的调节剂。
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