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一种利用多个生存数据集进行长期生存估计的贝叶斯分层混合治愈建模框架:来自未经治疗的转移性黑色素瘤的案例研究

A Bayesian Hierarchical Mixture Cure Modelling Framework to Utilize Multiple Survival Datasets for Long-Term Survivorship Estimates: A Case Study From Previously Untreated Metastatic Melanoma.

作者信息

Green Nathan, Kurt Murat, Moshyk Andriy, Larkin James, Baio Gianluca

机构信息

Department of Statistical Science, UCL, London, UK.

Worldwide Health Economics and Outcomes Research, Bristol Myers Squibb, Lawrence, New Jersey, USA.

出版信息

Stat Med. 2025 Jun;44(13-14):e70132. doi: 10.1002/sim.70132.

DOI:10.1002/sim.70132
PMID:40444748
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12124107/
Abstract

Time to an event of interest over a lifetime is a central measure of the clinical benefit of an intervention used in a health technology assessment (HTA). Within the same trial, multiple end-points may also be considered. For example, overall and progression-free survival time for different drugs in oncology studies. A common challenge is when an intervention is only effective for some proportion of the population who are not clinically identifiable. Therefore, latent group membership as well as separate survival models for identified groups need to be estimated. However, follow-up in trials may be relatively short leading to substantial censoring. We present a general Bayesian hierarchical framework that can handle this complexity by exploiting the similarity of cure fractions between end-points; accounting for the correlation between them and improving the extrapolation beyond the observed data. Assuming exchangeability between cure fractions facilitates the borrowing of information between end-points. We undertake a comprehensive simulation study to evaluate the model performance under different scenarios. We also show the benefits of using our approach with a motivating example, the CheckMate 067 phase 3 trial consisting of patients with metastatic melanoma treated with first line therapy.

摘要

一生中发生感兴趣事件的时间是卫生技术评估(HTA)中使用的干预措施临床益处的核心衡量指标。在同一试验中,也可能会考虑多个终点。例如,肿瘤学研究中不同药物的总生存期和无进展生存期。一个常见的挑战是,当一种干预措施仅对部分临床上无法识别的人群有效时。因此,需要估计潜在的组群归属以及已识别组群的单独生存模型。然而,试验中的随访可能相对较短,导致大量删失。我们提出了一个通用的贝叶斯分层框架,该框架可以通过利用终点之间治愈比例的相似性来处理这种复杂性;考虑它们之间的相关性,并改善对观测数据之外的推断。假设治愈比例之间具有可交换性有助于在终点之间借用信息。我们进行了一项全面的模拟研究,以评估不同场景下模型的性能。我们还通过一个具有启发性的例子展示了使用我们方法的益处,即由接受一线治疗的转移性黑色素瘤患者组成的CheckMate 067 3期试验。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/9a71bdf8802d/SIM-44-0-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/e1be1abe3f9b/SIM-44-0-g006.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/dbbb8a56bb0d/SIM-44-0-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/c859665bd4aa/SIM-44-0-g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/5388d1bfd1b5/SIM-44-0-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/82fdf8b97229/SIM-44-0-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/9a71bdf8802d/SIM-44-0-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/e1be1abe3f9b/SIM-44-0-g006.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/dbbb8a56bb0d/SIM-44-0-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/c859665bd4aa/SIM-44-0-g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/5388d1bfd1b5/SIM-44-0-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/82fdf8b97229/SIM-44-0-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0373/12124107/9a71bdf8802d/SIM-44-0-g003.jpg

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本文引用的文献

1
Stan: A Probabilistic Programming Language.斯坦:一种概率编程语言。
J Stat Softw. 2017;76. doi: 10.18637/jss.v076.i01. Epub 2017 Jan 11.
2
Blended Survival Curves: A New Approach to Extrapolation for Time-to-Event Outcomes from Clinical Trials in Health Technology Assessment.混合生存曲线:一种从健康技术评估临床试验中推断事件时间结局的新方法。
Med Decis Making. 2023 Apr;43(3):299-310. doi: 10.1177/0272989X221134545. Epub 2022 Oct 31.
3
Classifying information-sharing methods.分类信息共享方法。
BMC Med Res Methodol. 2021 May 22;21(1):107. doi: 10.1186/s12874-021-01292-z.
4
Evaluation of survival extrapolation in immuno-oncology using multiple pre-planned data cuts: learnings to aid in model selection.使用多次预先计划的数据切割评估免疫肿瘤学中的生存外推:辅助模型选择的经验教训。
BMC Med Res Methodol. 2020 May 6;20(1):103. doi: 10.1186/s12874-020-00997-x.
5
Five-Year Survival with Combined Nivolumab and Ipilimumab in Advanced Melanoma.纳武利尤单抗联合伊匹木单抗治疗晚期黑色素瘤的 5 年生存数据
N Engl J Med. 2019 Oct 17;381(16):1535-1546. doi: 10.1056/NEJMoa1910836. Epub 2019 Sep 28.
6
Estimating Lifetime Benefits Associated with Immuno-Oncology Therapies: Challenges and Approaches for Overall Survival Extrapolations.估算免疫肿瘤疗法相关的终生获益:总体生存外推的挑战与方法。
Pharmacoeconomics. 2019 Sep;37(9):1129-1138. doi: 10.1007/s40273-019-00806-4.
7
Combining Immune Checkpoint Inhibitors: Established and Emerging Targets and Strategies to Improve Outcomes in Melanoma.联合免疫检查点抑制剂:改善黑色素瘤治疗效果的既定和新兴靶点及策略。
Front Immunol. 2019 Mar 19;10:453. doi: 10.3389/fimmu.2019.00453. eCollection 2019.
8
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.
9
Nivolumab plus ipilimumab or nivolumab alone versus ipilimumab alone in advanced melanoma (CheckMate 067): 4-year outcomes of a multicentre, randomised, phase 3 trial.纳武利尤单抗联合伊匹单抗或纳武利尤单抗单药对比伊匹单抗单药治疗晚期黑色素瘤(CheckMate 067):一项多中心、随机、III 期临床试验的 4 年结果。
Lancet Oncol. 2018 Nov;19(11):1480-1492. doi: 10.1016/S1470-2045(18)30700-9. Epub 2018 Oct 22.
10
Bayesian Multiparameter Evidence Synthesis to Inform Decision Making: A Case Study in Metastatic Hormone-Refractory Prostate Cancer.贝叶斯多参数证据综合在决策制定中的应用:转移性激素难治性前列腺癌的案例研究。
Med Decis Making. 2018 Oct;38(7):834-848. doi: 10.1177/0272989X18788537. Epub 2018 Aug 13.