{"title":"学习最佳个性化治疗方案的稳健协变量平衡法","authors":"Canhui Li, Donglin Zeng, Wensheng Zhu","doi":"10.1093/biomet/asae036","DOIUrl":null,"url":null,"abstract":"Summary One of the most important problems in precision medicine is to find the optimal individualized treatment rule, which is designed to recommend treatment decisions and maximize overall clinical benefit to patients based on their individual characteristics. Typically, the expected clinical outcome is required to be estimated first, in which an outcome regression model or a propensity score model usually needs to be assumed for most of the existing statistical methods. However, if either model assumption is invalid, the estimated treatment regime is not reliable. In this article, we first define a contrast value function, which is the basis of the study for individualized treatment regimes. Then we construct a hybrid estimator of the contrast value function, by combining two types of estimation methods. We further propose a robust covariate-balancing estimator of the contrast value function by combining the inverse probability weighted method and matching method, which is based on the covariate balancing propensity score proposed by Imai and Ratkovic (2014). Theoretical results show that the proposed estimator is doubly robust, that is, it is consistent if either the propensity score model or the matching is correct. Based on a large number of simulation studies, we demonstrate that the proposed estimator outperforms existing methods. Lastly, the proposed method is illustrated through analysis of the SUPPORT study.","PeriodicalId":9001,"journal":{"name":"Biometrika","volume":null,"pages":null},"PeriodicalIF":2.4000,"publicationDate":"2024-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Robust Covariate-Balancing Method in Learning Optimal Individualized Treatment Regimes\",\"authors\":\"Canhui Li, Donglin Zeng, Wensheng Zhu\",\"doi\":\"10.1093/biomet/asae036\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Summary One of the most important problems in precision medicine is to find the optimal individualized treatment rule, which is designed to recommend treatment decisions and maximize overall clinical benefit to patients based on their individual characteristics. Typically, the expected clinical outcome is required to be estimated first, in which an outcome regression model or a propensity score model usually needs to be assumed for most of the existing statistical methods. However, if either model assumption is invalid, the estimated treatment regime is not reliable. In this article, we first define a contrast value function, which is the basis of the study for individualized treatment regimes. Then we construct a hybrid estimator of the contrast value function, by combining two types of estimation methods. We further propose a robust covariate-balancing estimator of the contrast value function by combining the inverse probability weighted method and matching method, which is based on the covariate balancing propensity score proposed by Imai and Ratkovic (2014). Theoretical results show that the proposed estimator is doubly robust, that is, it is consistent if either the propensity score model or the matching is correct. Based on a large number of simulation studies, we demonstrate that the proposed estimator outperforms existing methods. Lastly, the proposed method is illustrated through analysis of the SUPPORT study.\",\"PeriodicalId\":9001,\"journal\":{\"name\":\"Biometrika\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":2.4000,\"publicationDate\":\"2024-07-17\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Biometrika\",\"FirstCategoryId\":\"100\",\"ListUrlMain\":\"https://doi.org/10.1093/biomet/asae036\",\"RegionNum\":2,\"RegionCategory\":\"数学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"BIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Biometrika","FirstCategoryId":"100","ListUrlMain":"https://doi.org/10.1093/biomet/asae036","RegionNum":2,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"BIOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
摘要 精准医疗中最重要的问题之一是找到最佳个体化治疗规则,该规则旨在根据患者的个体特征推荐治疗决策,并使患者的总体临床获益最大化。通常情况下,首先需要估计预期临床结果,在此过程中,大多数现有统计方法通常需要假设结果回归模型或倾向评分模型。然而,如果任一模型假设无效,估计出的治疗方案就不可靠。在本文中,我们首先定义了对比值函数,这是研究个体化治疗方案的基础。然后,我们结合两种估计方法,构建了对比值函数的混合估计器。我们进一步结合反概率加权法和匹配法,在 Imai 和 Ratkovic(2014 年)提出的共变平衡倾向得分的基础上,提出了一种稳健的共变平衡对比值函数估计器。理论结果表明,所提出的估计器具有双重稳健性,即如果倾向得分模型或匹配正确,则估计器是一致的。基于大量的模拟研究,我们证明了所提出的估计方法优于现有方法。最后,我们通过对 SUPPORT 研究的分析来说明所提出的方法。
Robust Covariate-Balancing Method in Learning Optimal Individualized Treatment Regimes
Summary One of the most important problems in precision medicine is to find the optimal individualized treatment rule, which is designed to recommend treatment decisions and maximize overall clinical benefit to patients based on their individual characteristics. Typically, the expected clinical outcome is required to be estimated first, in which an outcome regression model or a propensity score model usually needs to be assumed for most of the existing statistical methods. However, if either model assumption is invalid, the estimated treatment regime is not reliable. In this article, we first define a contrast value function, which is the basis of the study for individualized treatment regimes. Then we construct a hybrid estimator of the contrast value function, by combining two types of estimation methods. We further propose a robust covariate-balancing estimator of the contrast value function by combining the inverse probability weighted method and matching method, which is based on the covariate balancing propensity score proposed by Imai and Ratkovic (2014). Theoretical results show that the proposed estimator is doubly robust, that is, it is consistent if either the propensity score model or the matching is correct. Based on a large number of simulation studies, we demonstrate that the proposed estimator outperforms existing methods. Lastly, the proposed method is illustrated through analysis of the SUPPORT study.
期刊介绍:
Biometrika is primarily a journal of statistics in which emphasis is placed on papers containing original theoretical contributions of direct or potential value in applications. From time to time, papers in bordering fields are also published.