候选代用终点与晚期胆道癌的总生存率的关联
Florian Castet1, Carles Fabregat-Franco2, John Bridgewater3
1Upper Gastrointestinal and Endocrine Tumor Unit, Vall d'Hebron Institute of Oncology (VHIO), Vall d'Hebron University Hospital, Barcelona, Spain.
Journal of hepatology
|June 5, 2025
概括
在胆道癌 (BTC) 试验中,无进展生存率 (PFS) 与整体生存率 (OS) 有适度的相关性. 客观反应率 (ORR) 和疾病控制率 (DCR) 的相关性较低,这表明在BTC研究中需要更好的替代终点.
科学领域:
- 在瘤学瘤学.
- 临床试验 临床试验
- 生物统计学 生物统计学
背景情况:
- 像PFS,ORR和DCR这样的替代终点越来越多地用于胆道癌症 (BTC) 试验,以加快药物批准和降低成本.
- 然而,这些替代终点与BTC的整体生存 (OS) 优势的相关性尚未完全确定.
- 这项研究研究了PFS,ORR和DCR与OS在试验和患者水平之间的关联.
研究的目的:
- 探索无进展生存率 (PFS),客观反应率 (ORR) 和疾病控制率 (DCR) 与胆道癌 (BTC) 的总生存率 (OS) 的相关性.
- 在临床试验和患者层面评估这些相关性.
- 为未来的BTC临床试验和监管评估选择适当的终点提供信息.
主要方法:
- 通过使用多个数据库和临床试验注册表,对高级BTC的II-III期试验进行了系统审查.
- 权重线性回归被用于试验级分析,以测量OS与PFS,ORR和DCR的相关性.
- 患者级分析包括来自五项随机试验和三项现实数据集的数据,分析不同患者队列之间的相关性.
主要成果:
- 对41项研究 (7,817名患者) 的试验级分析显示,PFS的R平方值为0.71,ORR为0.01,DCR为0.39.
- 在患者一级分析 (2506名患者) 中,PFS的相关系数在不同数据集中从0.73到0.86不等.
- 响应者分析发现客观响应率与整体存活率之间没有显著联系.
结论:
- 在胆道癌 (BTC) 中,在试验和患者水平上,无进展生存 (PFS) 与整体生存 (OS) 有中等的相关性.
- 客观反应率 (ORR) 和疾病控制率 (DCR) 与OS的相关性较低.
- 虽然PFS是BTC中OS目前最好的替代标记,但这些发现强调了开发和验证新型终点的关键需求.
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