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在纳入患者治疗选择的试验中,使用似然比检验分析生存结果。

Analysis of survival outcomes using likelihood ratio test in trials incorporating patient's treatment choice.

作者信息

Chahine Rouba A, Aban Inmaculada

机构信息

SSES-Analytics, RTI International, Research Triangle Park, NC, USA.

Department of Biostatistics, The University of Alabama at Birmingham, Birmingham, AL, USA.

出版信息

J Appl Stat. 2023 Apr 11;51(7):1344-1358. doi: 10.1080/02664763.2023.2199177. eCollection 2024.

Abstract

Methods for designing and analyzing multiple arms survival trials that incorporate patient's treatment choice are needed. In these trials, patients are randomized into two groups, random and choice. Participants in the choice group choose their treatment, which is not a current standard practice in randomized clinical trials. In this paper, we propose a new method based on the likelihood function to design and analyze these trials with time to event outcomes in the presence of non-informative right censoring. We use simulations to evaluate the methods for Weibull outcomes, complete and censored. Finally, we provide an illustration for designing a study in which we discuss some design considerations and demonstrate the methods.

摘要

需要设计和分析纳入患者治疗选择的多臂生存试验的方法。在这些试验中,患者被随机分为两组,即随机组和选择组。选择组的参与者选择他们的治疗方法,这在随机临床试验中不是当前的标准做法。在本文中,我们提出了一种基于似然函数的新方法,用于在存在非信息性右删失的情况下,设计和分析这些具有事件发生时间结局的试验。我们使用模拟来评估威布尔结局、完整数据和删失数据的方法。最后,我们提供了一个设计研究的示例,在其中我们讨论了一些设计考虑因素并演示了这些方法。

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引用本文的文献

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Likelihood based inferences for trials incorporating participant's treatment choice.纳入参与者治疗选择的试验的基于似然性的推断。
Contemp Clin Trials Commun. 2024 May 15;39:101306. doi: 10.1016/j.conctc.2024.101306. eCollection 2024 Jun.

本文引用的文献

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Using simulation studies to evaluate statistical methods.运用模拟研究评估统计方法。
Stat Med. 2019 May 20;38(11):2074-2102. doi: 10.1002/sim.8086. Epub 2019 Jan 16.
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The effect of patient choice of intervention on health outcomes.患者对干预措施的选择对健康结果的影响。
Contemp Clin Trials. 2008 Sep;29(5):679-86. doi: 10.1016/j.cct.2008.04.002. Epub 2008 Apr 20.
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Patient preferences in randomised trials: threat or opportunity?随机试验中的患者偏好:威胁还是机遇?
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