Qijia He, Shixiao Zhang, Michael L LeBlanc, Ying-Qi Zhao
{"title":"通过优化调整后的较长存活概率,估算个体化治疗规则。","authors":"Qijia He, Shixiao Zhang, Michael L LeBlanc, Ying-Qi Zhao","doi":"10.1177/09622802241262525","DOIUrl":null,"url":null,"abstract":"<p><p>Individualized treatment rules inform tailored treatment decisions based on the patient's information, where the goal is to optimize clinical benefit for the population. When the clinical outcome of interest is survival time, most of current approaches typically aim to maximize the expected time of survival. We propose a new criterion for constructing Individualized treatment rules that optimize the clinical benefit with survival outcomes, termed as the adjusted probability of a longer survival. This objective captures the likelihood of living longer with being on treatment, compared to the alternative, which provides an alternative and often straightforward interpretation to communicate with clinicians and patients. We view it as an alternative to the survival analysis standard of the hazard ratio and the increasingly used restricted mean survival time. We develop a new method to construct the optimal Individualized treatment rule by maximizing a nonparametric estimator of the adjusted probability of a longer survival for a decision rule. Simulation studies demonstrate the reliability of the proposed method across a range of different scenarios. We further perform data analysis using data collected from a randomized Phase III clinical trial (SWOG S0819).</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"1517-1530"},"PeriodicalIF":1.6000,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Estimating individualized treatment rules by optimizing the adjusted probability of a longer survival.\",\"authors\":\"Qijia He, Shixiao Zhang, Michael L LeBlanc, Ying-Qi Zhao\",\"doi\":\"10.1177/09622802241262525\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Individualized treatment rules inform tailored treatment decisions based on the patient's information, where the goal is to optimize clinical benefit for the population. When the clinical outcome of interest is survival time, most of current approaches typically aim to maximize the expected time of survival. We propose a new criterion for constructing Individualized treatment rules that optimize the clinical benefit with survival outcomes, termed as the adjusted probability of a longer survival. This objective captures the likelihood of living longer with being on treatment, compared to the alternative, which provides an alternative and often straightforward interpretation to communicate with clinicians and patients. We view it as an alternative to the survival analysis standard of the hazard ratio and the increasingly used restricted mean survival time. We develop a new method to construct the optimal Individualized treatment rule by maximizing a nonparametric estimator of the adjusted probability of a longer survival for a decision rule. Simulation studies demonstrate the reliability of the proposed method across a range of different scenarios. We further perform data analysis using data collected from a randomized Phase III clinical trial (SWOG S0819).</p>\",\"PeriodicalId\":22038,\"journal\":{\"name\":\"Statistical Methods in Medical Research\",\"volume\":\" \",\"pages\":\"1517-1530\"},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2024-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Statistical Methods in Medical Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/09622802241262525\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/7/25 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"HEALTH CARE SCIENCES & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Statistical Methods in Medical Research","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/09622802241262525","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/7/25 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
引用次数: 0
摘要
个体化治疗规则根据患者的信息提供量身定制的治疗决策,其目标是优化人群的临床获益。当关注的临床结果是存活时间时,目前的大多数方法通常以最大化预期存活时间为目标。我们提出了一种新的标准,用于构建个体化治疗规则,优化临床获益与生存结果,即调整后的延长生存概率。这一目标捕捉了与其他方法相比,接受治疗后存活时间更长的可能性,为临床医生和患者提供了另一种直截了当的解释。我们将其视为危险比这一生存分析标准和使用日益广泛的受限平均生存时间的替代方案。我们开发了一种新方法,通过最大化决策规则的调整后较长生存期概率的非参数估计来构建最佳个体化治疗规则。模拟研究证明了所提方法在各种不同情况下的可靠性。我们还利用从随机 III 期临床试验(SWOG S0819)中收集的数据进行了进一步的数据分析。
Estimating individualized treatment rules by optimizing the adjusted probability of a longer survival.
Individualized treatment rules inform tailored treatment decisions based on the patient's information, where the goal is to optimize clinical benefit for the population. When the clinical outcome of interest is survival time, most of current approaches typically aim to maximize the expected time of survival. We propose a new criterion for constructing Individualized treatment rules that optimize the clinical benefit with survival outcomes, termed as the adjusted probability of a longer survival. This objective captures the likelihood of living longer with being on treatment, compared to the alternative, which provides an alternative and often straightforward interpretation to communicate with clinicians and patients. We view it as an alternative to the survival analysis standard of the hazard ratio and the increasingly used restricted mean survival time. We develop a new method to construct the optimal Individualized treatment rule by maximizing a nonparametric estimator of the adjusted probability of a longer survival for a decision rule. Simulation studies demonstrate the reliability of the proposed method across a range of different scenarios. We further perform data analysis using data collected from a randomized Phase III clinical trial (SWOG S0819).
期刊介绍:
Statistical Methods in Medical Research is a peer reviewed scholarly journal and is the leading vehicle for articles in all the main areas of medical statistics and an essential reference for all medical statisticians. This unique journal is devoted solely to statistics and medicine and aims to keep professionals abreast of the many powerful statistical techniques now available to the medical profession. This journal is a member of the Committee on Publication Ethics (COPE)