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将患者报告的结果纳入剂量确定临床试验,并持续招募患者

Anaïs Andrillon^1,2, Lucie Biard¹, Shing M Lee³

¹INSERM U1153 Team ECSTRRA, Université Paris Cité, Paris, France.

Journal of biopharmaceutical statistics

|July 27, 2023

概括

新的方法,TITE-PRO-CRM和TITE-CRM+PRO,通过结合患者报告的结果 (PRO) 和临床医生毒性数据来提高瘤学剂量确定试验,以更有效地估计最大耐受剂量 (MTD).

科学领域:

临床试验临床试验
在瘤学瘤学.
生物统计学生物统计学

背景情况:

瘤学中的剂量确定试验传统上依赖于临床医生评估的毒性来确定最大耐受剂量 (MTD).
患者报告的结果 (PROs) 提供了对治疗耐受性的宝贵见解,但缺乏用于剂量分配的标准化方法.
持续再评估PRO方法 (PRO-CRM) 是为了将PRO纳入剂量确定研究而开发的.

研究的目的:

为剂量确定试验提出和评估两个扩展的PRO-CRM方法 (TITE-PRO-CRM和TITE-CRM+PRO).
纳入持续的患者注册和扩展的毒性观察窗口,以捕捉晚发病或累积的毒性.
使用加权概率方法,以纳入部分毒性后续信息.

主要方法:

在整个试验中,TITE-PRO-CRM集成了PRO和临床医生的数据,用于剂量分配和MTD估计.
TITE-CRM+PRO使用临床医生数据进行试验期间的剂量分配,并使用PRO数据进行最终的MTD估计.
进行了模拟研究,以在各种场景下比较拟议方法与PRO-CRM的性能.

主要成果:

TITE-PRO-CRM证明了与PRO-CRM可比的剂量建议和分配,可能将试验持续时间减半.
TITE-CRM+PRO的性能略低,但在更大的样本大小下可以达到类似的有效性.
这两种方法在较高的积累率,不同的毒性危险和相关的毒性数据方面都被证明是可靠的.

结论:

拟议的TITE-PRO-CRM和TITE-CRM+PRO为瘤学剂量检测试验提供了高效和强大的方法.
这些方法有效地整合了患者报告的结果和临床医生的评估,以改善MTD的确定.
扩展的观察窗口和加权的概率方法提高了复杂的毒性模式的捕获.

关键词:

加快剂量发现速度.贝叶斯的自适应剂量发现.持续重新评估的方法.晚期发生的毒性.多重毒性制约因素多重毒性制约因素部分毒性随访部分毒性随访第一个阶段的试验试验.时间到事件的时间.

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