Estimating Optimal Dynamic Treatment Regimes With Survival Outcomes

2020
The statistical study of precision medicine is concerned with dynamic treatment regimes (DTRs) in which treatment decisions are tailored to patient-level information. Individuals are followed through multiple stages of clinical intervention, and the goal is to perform inferences on the sequence of personalized treatment decision rules to be applied in practice. Of interest is the identification of an optimal DTR, that is, the sequence of treatment decisions that yields the best expected outcome. Statistical methods for identifying optimal DTRs from observational data are theoretically complex and not easily implementable by researchers, especially when the outcome of interest is survival time. We propose a doubly robust, easy to implement method for estimating optimal DTRs with survival endpoints subject to right-censoring which requires solving a series of weighted generalized estimating equations. We provide a proof of consistency that relies on the balancing property of the weights and derive a formula for the asymptotic variance of the resulting estimators. We illustrate our novel approach with an application to the treatment of rheumatoid arthritis using observational data from the Scottish Early Rheumatoid Arthritis Inception Cohort. Our method, called dynamic weighted survival modeling, has been implemented in the DTRreg R package. for this article are available online.
JOURNAL OF THE AMERICAN STATISTICAL ASSOCIATION
页码:1531-1539|卷号:115|期号:531
ISSN:0162-1459
收录类型
SSCI
发表日期
2020
学科领域
循证社会科学-方法
国家
加拿大
语种
英语
DOI
10.1080/01621459.2019.1629939
其他关键词
ERYTHROCYTE SEDIMENTATION-RATE; C-REACTIVE PROTEIN; MODELS
EISSN
1537-274X
资助机构
Fonds de recherche du Quebec Nature et Technologies [199803]; Translational Medicine Research Collaboration, a consortiummade up of the University of Aberdeen [INF-GU-168]; Translational Medicine Research Collaboration, a consortiummade up of the University of Dundee [INF-GU-168]; Translational Medicine Research Collaboration, a consortiummade up of the University of Edinburgh [INF-GU-168]; Translational Medicine Research Collaboration, a consortiummade up of the University of Glasgow [INF-GU-168]; NHS Health Board (Grampian) [INF-GU-168]; NHS Health Board (Tayside) [INF-GU-168]; NHS Health Board (Lothian) [INF-GU-168]; NHS Health Board (Greater Glasgow Clyde) [INF-GU-168]; PfizerPfizer [INF-GU-168]; Chief Scientific Office [ETM-40]
资助信息
This work was supported by a doctoral scholarship from the Fonds de recherche du Quebec Nature et Technologies (Ref. 199803) and by awards (INF-GU-168) from (1) the Translational Medicine Research Collaboration, a consortiummade up of the Universities of Aberdeen, Dundee, Edinburgh and Glasgow, the four associated NHS Health Boards (Grampian, Tayside, Lothian and Greater Glasgow & Clyde), and Pfizer and (2) from the Chief Scientific Office (Ref ETM-40).
被引频次(WOS)
10
被引更新日期
2022-01
来源机构
McGill University University of Cambridge
关键词
Accelerated failure time model Balancing weights Causal inference Censored data Precision medicine Rheumatoid arthritis