{"title":"利用区间失效时间数据提高病例队列研究的估算效率。","authors":"Qingning Zhou, Kin Yau Wong","doi":"10.1177/09622802241268601","DOIUrl":null,"url":null,"abstract":"<p><p>The case-cohort design is a commonly used cost-effective sampling strategy for large cohort studies, where some covariates are expensive to measure or obtain. In this paper, we consider regression analysis under a case-cohort study with interval-censored failure time data, where the failure time is only known to fall within an interval instead of being exactly observed. A common approach to analyzing data from a case-cohort study is the inverse probability weighting approach, where only subjects in the case-cohort sample are used in estimation, and the subjects are weighted based on the probability of inclusion into the case-cohort sample. This approach, though consistent, is generally inefficient as it does not incorporate information outside the case-cohort sample. To improve efficiency, we first develop a sieve maximum weighted likelihood estimator under the Cox model based on the case-cohort sample and then propose a procedure to update this estimator by using information in the full cohort. We show that the update estimator is consistent, asymptotically normal, and at least as efficient as the original estimator. The proposed method can flexibly incorporate auxiliary variables to improve estimation efficiency. A weighted bootstrap procedure is employed for variance estimation. Simulation results indicate that the proposed method works well in practical situations. An application to a Phase 3 HIV vaccine efficacy trial is provided for illustration.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"1673-1685"},"PeriodicalIF":1.6000,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Improving estimation efficiency of case-cohort studies with interval-censored failure time data.\",\"authors\":\"Qingning Zhou, Kin Yau Wong\",\"doi\":\"10.1177/09622802241268601\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>The case-cohort design is a commonly used cost-effective sampling strategy for large cohort studies, where some covariates are expensive to measure or obtain. In this paper, we consider regression analysis under a case-cohort study with interval-censored failure time data, where the failure time is only known to fall within an interval instead of being exactly observed. A common approach to analyzing data from a case-cohort study is the inverse probability weighting approach, where only subjects in the case-cohort sample are used in estimation, and the subjects are weighted based on the probability of inclusion into the case-cohort sample. This approach, though consistent, is generally inefficient as it does not incorporate information outside the case-cohort sample. To improve efficiency, we first develop a sieve maximum weighted likelihood estimator under the Cox model based on the case-cohort sample and then propose a procedure to update this estimator by using information in the full cohort. We show that the update estimator is consistent, asymptotically normal, and at least as efficient as the original estimator. The proposed method can flexibly incorporate auxiliary variables to improve estimation efficiency. A weighted bootstrap procedure is employed for variance estimation. Simulation results indicate that the proposed method works well in practical situations. An application to a Phase 3 HIV vaccine efficacy trial is provided for illustration.</p>\",\"PeriodicalId\":22038,\"journal\":{\"name\":\"Statistical Methods in Medical Research\",\"volume\":\" \",\"pages\":\"1673-1685\"},\"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/10.1177/09622802241268601\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/8/6 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/10.1177/09622802241268601","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/8/6 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
引用次数: 0
摘要
病例队列设计是大型队列研究中常用的一种具有成本效益的抽样策略,因为有些协变量的测量或获取成本较高。在本文中,我们将考虑在病例队列研究中使用区间删失的故障时间数据进行回归分析,在区间删失的故障时间中,只知道故障时间在一个区间内,而不是精确观测到的故障时间。分析病例队列研究数据的常用方法是反概率加权法,即只使用病例队列样本中的受试者进行估计,并根据纳入病例队列样本的概率对受试者进行加权。这种方法虽然前后一致,但由于没有纳入病例队列样本以外的信息,因此效率普遍较低。为了提高效率,我们首先开发了基于病例队列样本的考克斯模型下的筛网最大加权似然估计器,然后提出了一种利用完整队列信息更新该估计器的程序。我们的研究表明,更新后的估计值是一致的、渐近正态的,并且至少与原始估计值一样有效。所提出的方法可以灵活地结合辅助变量来提高估计效率。方差估计采用了加权自举程序。模拟结果表明,所提出的方法在实际情况中效果良好。本文还提供了一个 HIV 疫苗 3 期疗效试验的应用实例,以资说明。
Improving estimation efficiency of case-cohort studies with interval-censored failure time data.
The case-cohort design is a commonly used cost-effective sampling strategy for large cohort studies, where some covariates are expensive to measure or obtain. In this paper, we consider regression analysis under a case-cohort study with interval-censored failure time data, where the failure time is only known to fall within an interval instead of being exactly observed. A common approach to analyzing data from a case-cohort study is the inverse probability weighting approach, where only subjects in the case-cohort sample are used in estimation, and the subjects are weighted based on the probability of inclusion into the case-cohort sample. This approach, though consistent, is generally inefficient as it does not incorporate information outside the case-cohort sample. To improve efficiency, we first develop a sieve maximum weighted likelihood estimator under the Cox model based on the case-cohort sample and then propose a procedure to update this estimator by using information in the full cohort. We show that the update estimator is consistent, asymptotically normal, and at least as efficient as the original estimator. The proposed method can flexibly incorporate auxiliary variables to improve estimation efficiency. A weighted bootstrap procedure is employed for variance estimation. Simulation results indicate that the proposed method works well in practical situations. An application to a Phase 3 HIV vaccine efficacy trial is provided for illustration.
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)