在随机临床试验中检测治疗模式的适当性,对长期幸存者的随机临床试验
Cheryl Kouadio1,2,3, Subodh Selukar4, Megan Othus5
1INSERM U1342, Team ECSTRRA, Hôpital Saint Louis, Paris, France.
JCO clinical cancer informatics
|December 15, 2025
概括
治愈模型适用于分析随机临床试验 (RCT) 中的长期幸存者的生存数据. 对每个手臂单独应用的审查治愈受试者的比率估计 (RECeUS) 方法有助于确认治愈患者的存在.
科学领域:
- 生物统计学 生物统计学
- 临床试验方法论 临床试验方法论
- 生存分析的分析.
背景情况:
- 在长期幸存者随机临床试验 (RCT) 中分析正确审查的终点存在挑战.
- 长期幸存者的存在可能表明患者群体中的"治愈"部分.
- 在这种情况下,传统的生存模型可能无法充分捕捉治愈的动态.
研究的目的:
- 评估治愈模型的适用性,以分析长期幸存者的恶性瘤RCT的右控终点.
- 扩大对被审查治愈受试者的比率估计 (RECeUS) 方法用于RCT.
主要方法:
- 根据RECeUS方法制定了四个决策规则,以评估RCT中治疗模式的适当性.
- 一项模拟研究评估了这些规则的性能和链接功能的影响.
- 拟议的方法是使用来自急性白血病和COVID-19的RCT的现实数据来说明的.
主要成果:
- 模拟结果表明,将RECeUS标准应用于至少一个随机分组是各种治疗效果场景中最强大的决策规则.
- 治疗模式被认为适用于分析的RCT数据,无论采用什么具体的决策规则.
- 在RCT环境中,RECeUS方法证明了在确认治愈患者的存在方面具有实用性.
结论:
- 在RCT中生存曲线的平原表明治愈模型的潜在适当性.
- 应将RECeUS方法独立应用于每个随机分组,以验证治愈人口的存在.
- 在至少一个随机分组中满足RECeUS标准支持在RCT生存分析中使用治愈模型.
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