加权反向计数过程(WRCP):一种新的方法,通过自适应加权来量化多个时间到事件结果的总体治疗效果。

IF 1.6 3区 医学 Q3 HEALTH CARE SCIENCES & SERVICES Statistical Methods in Medical Research Pub Date : 2025-01-01 Epub Date: 2024-12-04 DOI:10.1177/09622802241298702
Qianmiao Gao, Wei Zhong
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引用次数: 0

摘要

在纵向随机研究中,收集多个事件发生时间的结果,可以通过一个复合终点来量化总体治疗效果,该复合终点定义为任何选定事件(包括死亡)首次发生的时间。最近提出了反向计数过程(RCP),以延长限制平均生存时间(RMST)方法,其优势是利用“首次发生”终点以外的事件观察。然而,这种解释可能是有问题的,因为RCP对所有事件一视同仁,而不考虑它们与总体生存的不同关系。在这项工作中,我们提出了一种新的方法,加权反向计数过程(WRCP),以构建加权复合终点来评估整体治疗效果。采用多状态转移模型对事件之间的关联进行建模,并利用试验数据,基于非致命终点与死亡之间的关联,开发了一种自适应加权算法来确定各个终点的权重。通过仿真研究,比较了WRCP与RCP、log-rank检验和RMST方法的性能。结果表明,WRCP是一种强大的鲁棒方法,可以检测整体治疗效果,同时很好地控制不同模拟场景下的临床假阳性率。
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Weighted reverse counting process (WRCP): A novel approach to quantify the overall treatment effect with multiple time-to-event outcomes by adaptive weighting.

In a longitudinal randomized study where multiple time-to-event outcomes are collected, the overall treatment effect may be quantified by a composite endpoint defined as the time to the first occurrence of any of the selected events including death. The reverse counting process (RCP) was recently proposed to extend the restricted mean survival time (RMST) approach with an advantage of utilizing observations of events beyond the "first-occurrence" endpoint. However, the interpretation may be questionable because RCP treats all events equally without considering their different associations with the overall survival. In this work, we propose a novel approach, the weighted reverse counting process (WRCP), to construct a weighted composite endpoint to evaluate the overall treatment effect. A multi-state transition model is used to model the association between events, and an adaptive weighting algorithm is developed to determine the weight for individual endpoints based on the association between the nonfatal endpoints and death using the trial data. Simulation studies are presented to compare the performance of WRCP with RCP, log-rank test and RMST approach. The results show that WRCP is a powerful and robust method to detect the overall treatment effect while controlling the clinically false positive rate well across different simulation scenarios.

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来源期刊
Statistical Methods in Medical Research
Statistical Methods in Medical Research 医学-数学与计算生物学
CiteScore
4.10
自引率
4.30%
发文量
127
审稿时长
>12 weeks
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)
期刊最新文献
Extension of Fisher's least significant difference method to multi-armed group-sequential response-adaptive designs. Generalized framework for identifying meaningful heterogenous treatment effects in observational studies: A parametric data-adaptive G-computation approach. The relative efficiency of staircase and stepped wedge cluster randomised trial designs. Bayesian mixture models for phylogenetic source attribution from consensus sequences and time since infection estimates. Jointly assessing multiple endpoints in pilot and feasibility studies.
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