Bayesian Nonparametric Model for Heterogeneous Treatment Effects With Zero-Inflated Data.

IF 1.8 4区 医学 Q3 MATHEMATICAL & COMPUTATIONAL BIOLOGY Statistics in Medicine Pub Date : 2024-12-30 Epub Date: 2024-11-28 DOI:10.1002/sim.10266
Chanmin Kim, Yisheng Li, Ting Xu, Zhongxing Liao
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Abstract

One goal of precision medicine is to develop effective treatments for patients by tailoring to their individual demographic, clinical, and/or genetic characteristics. To achieve this goal, statistical models must be developed that can identify and evaluate potentially heterogeneous treatment effects in a robust manner. The oft-cited existing methods for assessing treatment effect heterogeneity are based upon parametric models with interactions or conditioning on covariate values, the performance of which is sensitive to the omission of important covariates and/or the choice of their values. We propose a new Bayesian nonparametric (BNP) method for estimating heterogeneous causal effects in studies with zero-inflated outcome data, which arise commonly in health-related studies. We employ the enriched Dirichlet process (EDP) mixture in our BNP approach, establishing a connection between an outcome DP mixture and a covariate DP mixture. This enables us to estimate posterior distributions concurrently, facilitating flexible inference regarding individual causal effects. We show in a set of simulation studies that the proposed method outperforms two other BNP methods in terms of bias and mean squared error (MSE) of the conditional average treatment effect estimates. In particular, the proposed model has the advantage of appropriately reflecting uncertainty in regions where the overlap condition is violated compared to other competing models. We apply the proposed method to a study of the relationship between heart radiation dose parameters and the blood level of high-sensitivity cardiac troponin T (hs-cTnT) to examine if the effect of a high mean heart radiation dose on hs-cTnT varies by baseline characteristics.

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零膨胀数据下异质治疗效果的贝叶斯非参数模型
精准医疗的目标之一是根据患者的人口统计、临床和/或遗传特征量身定制有效的治疗方法。为了实现这一目标,必须开发能够以稳健的方式识别和评估潜在异质性治疗效果的统计模型。经常被引用的评估治疗效果异质性的现有方法是基于协变量值的相互作用或条件作用的参数模型,其性能对重要协变量的遗漏和/或其值的选择很敏感。我们提出了一种新的贝叶斯非参数(BNP)方法来估计零膨胀结局数据研究中的异质性因果效应,这在与健康相关的研究中很常见。我们在BNP方法中采用了富狄利克雷过程(EDP)混合物,建立了结果DP混合物和协变量DP混合物之间的联系。这使我们能够同时估计后验分布,促进对个体因果效应的灵活推断。我们在一组模拟研究中表明,所提出的方法在条件平均处理效果估计的偏差和均方误差(MSE)方面优于其他两种BNP方法。特别是,与其他竞争模型相比,该模型具有适当反映违反重叠条件区域的不确定性的优点。我们将提出的方法应用于心脏辐射剂量参数与高敏心肌肌钙蛋白T (hs-cTnT)血液水平之间的关系研究,以检查高平均心脏辐射剂量对hs-cTnT的影响是否因基线特征而异。
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来源期刊
Statistics in Medicine
Statistics in Medicine 医学-公共卫生、环境卫生与职业卫生
CiteScore
3.40
自引率
10.00%
发文量
334
审稿时长
2-4 weeks
期刊介绍: The journal aims to influence practice in medicine and its associated sciences through the publication of papers on statistical and other quantitative methods. Papers will explain new methods and demonstrate their application, preferably through a substantive, real, motivating example or a comprehensive evaluation based on an illustrative example. Alternatively, papers will report on case-studies where creative use or technical generalizations of established methodology is directed towards a substantive application. Reviews of, and tutorials on, general topics relevant to the application of statistics to medicine will also be published. The main criteria for publication are appropriateness of the statistical methods to a particular medical problem and clarity of exposition. Papers with primarily mathematical content will be excluded. The journal aims to enhance communication between statisticians, clinicians and medical researchers.
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