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一种用于治疗推荐的无参考 R-learner。

A reference-free R-learner for treatment recommendation.

机构信息

Design and Inovation, 7129Amgen Inc., Thousand Oaks, CA, USA.

Department of Biostatistics, 12284University of Nebraska Medical Center, Omaha, NE, USA.

出版信息

Stat Methods Med Res. 2023 Feb;32(2):404-424. doi: 10.1177/09622802221144326. Epub 2022 Dec 20.

Abstract

Assigning optimal treatments to individual patients based on their characteristics is the ultimate goal of precision medicine. Deriving evidence-based recommendations from observational data while considering the causal treatment effects and patient heterogeneity is a challenging task, especially in situations of multiple treatment options. Herein, we propose a reference-free R-learner based on a simplex algorithm for treatment recommendation. We showed through extensive simulation that the proposed method produced accurate recommendations that corresponded to optimal treatment outcomes, regardless of the reference group. We used the method to analyze data from the Systolic Blood Pressure Intervention Trial (SPRINT) and achieved recommendations consistent with the current clinical guidelines.

摘要

基于患者特征为个体患者分配最佳治疗方案是精准医学的最终目标。从观察性数据中得出基于证据的推荐意见,同时考虑因果治疗效果和患者异质性,是一项具有挑战性的任务,尤其是在存在多种治疗选择的情况下。在此,我们提出了一种基于单纯形算法的无参考 R-learner 用于治疗推荐。我们通过广泛的模拟表明,无论参考组如何,所提出的方法都能产生准确的推荐,这些推荐与最佳治疗效果相对应。我们使用该方法分析了收缩压干预试验(SPRINT)的数据,并得出了与当前临床指南一致的推荐意见。

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A reference-free R-learner for treatment recommendation.一种用于治疗推荐的无参考 R-learner。
Stat Methods Med Res. 2023 Feb;32(2):404-424. doi: 10.1177/09622802221144326. Epub 2022 Dec 20.
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本文引用的文献

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Robust Estimation of Heterogeneous Treatment Effects: An Algorithm-based Approach.异质性治疗效果的稳健估计:一种基于算法的方法。
Commun Stat Simul Comput. 2023;52(10):4981-4998. doi: 10.1080/03610918.2021.1974883. Epub 2021 Sep 14.
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Bayesian causal inference: a critical review.贝叶斯因果推断:批判性回顾。
Philos Trans A Math Phys Eng Sci. 2023 May 15;381(2247):20220153. doi: 10.1098/rsta.2022.0153. Epub 2023 Mar 27.
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Selective inference for effect modification via the lasso.通过套索进行效应修正的选择性推断。
J R Stat Soc Series B Stat Methodol. 2022 Apr;84(2):382-413. doi: 10.1111/rssb.12483. Epub 2021 Dec 14.
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A model-based multithreshold method for subgroup identification.基于模型的亚组识别多阈值方法。
Stat Med. 2019 Jun 30;38(14):2605-2631. doi: 10.1002/sim.8136. Epub 2019 Mar 18.
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Metalearners for estimating heterogeneous treatment effects using machine learning.使用机器学习估计异质处理效应的元学习器。
Proc Natl Acad Sci U S A. 2019 Mar 5;116(10):4156-4165. doi: 10.1073/pnas.1804597116. Epub 2019 Feb 15.

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