{"title":"利用工具变量对生存数据的最佳治疗方案进行双稳健估计","authors":"Xia Junwen, Zhan Zishu, Zhang Jingxiao","doi":"10.1007/s11222-024-10407-7","DOIUrl":null,"url":null,"abstract":"<p>In survival contexts, substantial literature exists on estimating optimal treatment regimes, where treatments are assigned based on personal characteristics to maximize the survival probability. These methods assume that a set of covariates is sufficient to deconfound the treatment-outcome relationship. However, this assumption can be limited in observational studies or randomized trials in which non-adherence occurs. Therefore, we propose a novel approach to estimating optimal treatment regimes when certain confounders are unobservable and a binary instrumental variable is available. Specifically, via a binary instrumental variable, we propose a semiparametric estimator for optimal treatment regimes by maximizing a Kaplan–Meier-like estimator of the survival function. Furthermore, to increase resistance to model misspecification, we construct novel doubly robust estimators. Since the estimators of the survival function are jagged, we incorporate kernel smoothing methods to improve performance. Under appropriate regularity conditions, the asymptotic properties are rigorously established. Moreover, the finite sample performance is evaluated through simulation studies. Finally, we illustrate our method using data from the National Cancer Institute’s prostate, lung, colorectal, and ovarian cancer screening trial.</p>","PeriodicalId":22058,"journal":{"name":"Statistics and Computing","volume":"153 1","pages":""},"PeriodicalIF":1.6000,"publicationDate":"2024-03-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Doubly robust estimation of optimal treatment regimes for survival data using an instrumental variable\",\"authors\":\"Xia Junwen, Zhan Zishu, Zhang Jingxiao\",\"doi\":\"10.1007/s11222-024-10407-7\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>In survival contexts, substantial literature exists on estimating optimal treatment regimes, where treatments are assigned based on personal characteristics to maximize the survival probability. These methods assume that a set of covariates is sufficient to deconfound the treatment-outcome relationship. However, this assumption can be limited in observational studies or randomized trials in which non-adherence occurs. Therefore, we propose a novel approach to estimating optimal treatment regimes when certain confounders are unobservable and a binary instrumental variable is available. Specifically, via a binary instrumental variable, we propose a semiparametric estimator for optimal treatment regimes by maximizing a Kaplan–Meier-like estimator of the survival function. Furthermore, to increase resistance to model misspecification, we construct novel doubly robust estimators. Since the estimators of the survival function are jagged, we incorporate kernel smoothing methods to improve performance. Under appropriate regularity conditions, the asymptotic properties are rigorously established. Moreover, the finite sample performance is evaluated through simulation studies. Finally, we illustrate our method using data from the National Cancer Institute’s prostate, lung, colorectal, and ovarian cancer screening trial.</p>\",\"PeriodicalId\":22058,\"journal\":{\"name\":\"Statistics and Computing\",\"volume\":\"153 1\",\"pages\":\"\"},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2024-03-16\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Statistics and Computing\",\"FirstCategoryId\":\"100\",\"ListUrlMain\":\"https://doi.org/10.1007/s11222-024-10407-7\",\"RegionNum\":2,\"RegionCategory\":\"数学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"COMPUTER SCIENCE, THEORY & METHODS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Statistics and Computing","FirstCategoryId":"100","ListUrlMain":"https://doi.org/10.1007/s11222-024-10407-7","RegionNum":2,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"COMPUTER SCIENCE, THEORY & METHODS","Score":null,"Total":0}
Doubly robust estimation of optimal treatment regimes for survival data using an instrumental variable
In survival contexts, substantial literature exists on estimating optimal treatment regimes, where treatments are assigned based on personal characteristics to maximize the survival probability. These methods assume that a set of covariates is sufficient to deconfound the treatment-outcome relationship. However, this assumption can be limited in observational studies or randomized trials in which non-adherence occurs. Therefore, we propose a novel approach to estimating optimal treatment regimes when certain confounders are unobservable and a binary instrumental variable is available. Specifically, via a binary instrumental variable, we propose a semiparametric estimator for optimal treatment regimes by maximizing a Kaplan–Meier-like estimator of the survival function. Furthermore, to increase resistance to model misspecification, we construct novel doubly robust estimators. Since the estimators of the survival function are jagged, we incorporate kernel smoothing methods to improve performance. Under appropriate regularity conditions, the asymptotic properties are rigorously established. Moreover, the finite sample performance is evaluated through simulation studies. Finally, we illustrate our method using data from the National Cancer Institute’s prostate, lung, colorectal, and ovarian cancer screening trial.
期刊介绍:
Statistics and Computing is a bi-monthly refereed journal which publishes papers covering the range of the interface between the statistical and computing sciences.
In particular, it addresses the use of statistical concepts in computing science, for example in machine learning, computer vision and data analytics, as well as the use of computers in data modelling, prediction and analysis. Specific topics which are covered include: techniques for evaluating analytically intractable problems such as bootstrap resampling, Markov chain Monte Carlo, sequential Monte Carlo, approximate Bayesian computation, search and optimization methods, stochastic simulation and Monte Carlo, graphics, computer environments, statistical approaches to software errors, information retrieval, machine learning, statistics of databases and database technology, huge data sets and big data analytics, computer algebra, graphical models, image processing, tomography, inverse problems and uncertainty quantification.
In addition, the journal contains original research reports, authoritative review papers, discussed papers, and occasional special issues on particular topics or carrying proceedings of relevant conferences. Statistics and Computing also publishes book review and software review sections.