Efficient estimation of the cox model when incorporating the subgroup restricted mean survival time.

IF 1.2 4区 医学 Q4 PHARMACOLOGY & PHARMACY Journal of Biopharmaceutical Statistics Pub Date : 2025-03-13 DOI:10.1080/10543406.2024.2444242
Jo-Ying Hung, Junjiang Zhong, Huang-Tz Ou, Pei-Fang Su
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Abstract

The restricted mean survival time has been widely used in the field of medical research because of its clear physical and simple clinical interpretation. In this paper, we propose an efficient estimation that incorporates the auxiliary restricted mean survival information into the estimation of the proportional hazard (PH) model. Compared to conventional models that do not incorporate available auxiliary information, the proposed method improves efficiency in estimating regression parameters by utilizing the double empirical likelihood method. We prove that the estimator asymptotically follows a multivariate normal distribution with a covariance matrix that can be consistently estimated. To address scenarios where the PH assumption is violated, we also extended the method to the stratified Cox model. In addition, simulation studies show that the proposed estimators are more efficient than those derived from the conventional partial likelihood approach. A type 2 diabetes dataset is then used to evaluate the risk of antidiabetic drugs and demonstrate the proposed method.

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当纳入亚组限制平均生存时间时,cox模型的有效估计。
有限平均生存时间因其明确的物理意义和简单的临床解释而广泛应用于医学研究领域。在本文中,我们提出了一种有效的估计,将辅助限制平均生存信息纳入比例风险(PH)模型的估计中。与传统模型不考虑可用辅助信息相比,该方法利用双经验似然法提高了回归参数估计的效率。我们证明了估计量渐近地服从一个具有可一致估计的协方差矩阵的多元正态分布。为了解决PH假设被违反的情况,我们还将该方法扩展到分层Cox模型。此外,仿真研究表明,所提出的估计量比传统的部分似然方法的估计量更有效。然后使用2型糖尿病数据集来评估抗糖尿病药物的风险并验证所提出的方法。
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来源期刊
Journal of Biopharmaceutical Statistics
Journal of Biopharmaceutical Statistics 医学-统计学与概率论
CiteScore
2.50
自引率
18.20%
发文量
71
审稿时长
6-12 weeks
期刊介绍: The Journal of Biopharmaceutical Statistics, a rapid publication journal, discusses quality applications of statistics in biopharmaceutical research and development. Now publishing six times per year, it includes expositions of statistical methodology with immediate applicability to biopharmaceutical research in the form of full-length and short manuscripts, review articles, selected/invited conference papers, short articles, and letters to the editor. Addressing timely and provocative topics important to the biostatistical profession, the journal covers: Drug, device, and biological research and development; Drug screening and drug design; Assessment of pharmacological activity; Pharmaceutical formulation and scale-up; Preclinical safety assessment; Bioavailability, bioequivalence, and pharmacokinetics; Phase, I, II, and III clinical development including complex innovative designs; Premarket approval assessment of clinical safety; Postmarketing surveillance; Big data and artificial intelligence and applications.
期刊最新文献
RETRACTED ARTICLE: A Bayesian joint model for multivariate longitudinal and time-to-event data with application to ALL maintenance studies. Correction. Statement of Retraction: A Bayesian joint model for multivariate longitudinal and time-to-event data with application to ALL maintenance studies. A constrained optimum adaptive design for dose finding in early phase clinical trials. MOVER tests for non-inferiority of the difference between two binary-outcome treatments in the matched-pairs design.
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