使用患者报告结果的同位素I期癌症临床试验设计
1Department of Biostatistics, Virginia Commonwealth University, Richmond, Virginia, U.S.A.
Statistics in biopharmaceutical research
|August 22, 2025
概括
这项研究引入了I期临床试验的新适应性设计,即患者报告的结果同位素 (PRO- ISO) 设计,该设计使用患者报告的结果安全地分配剂量并确定最大耐受剂量 (MTD). 在模拟中,PRO-ISO设计的性能与现有方法相美.
科学领域:
- 临床试验设计
- 药量测量
- 生物统计学
背景情况:
- 一期临床试验对于确定安全有效的药物剂量至关重要.
- 传统方法可能无法完全捕捉患者的经验或有效地适应毒性信号.
- 整合患者报告的结果 (PROs) 提供了更加以患者为中心的剂量升级方法.
研究的目的:
- 引入和评估一种新的适应性第一期临床试验设计,即患者报告的结果同位素 (PRO-ISO) 设计.
- 将PRO-ISO设计的性能与已知的方法进行比较,例如患者报告结果的持续再评估方法 (PRO-CRM).
- 扩展PRO-ISO方法以解决临床试验中晚期出现的毒性.
主要方法:
- 为第一阶段试验开发PRO-ISO适应性设计.
- 模拟研究以评估操作特征,包括剂量分配和目标剂量建议.
- 与PRO-CRM进行比较,并对PRO设置进行非参数最佳基准.
- 方法的扩展包括晚期出现的毒性.
主要成果:
- PRO-ISO设计有效地将参与者分配到可容忍的剂量中,根据观察到的患者报告的结果毒性事件进行调整.
- 模拟结果表明,在剂量推和患者分配方面,PRO- ISO的性能与PRO- CRM相当.
- 在PRO设置中,PRO-ISO与非参数最佳基准相比表现强.
结论:
- PRO- ISO设计为I期临床试验提供了灵活有效的方法,提高了患者的安全性和剂量确定.
- 这种新的设计强大地整合了患者报告的结果,为药物耐受性提供了有价值的见解.
- 该方法可适应复杂的情景,包括晚期出现的毒性,从而提高其临床适用性.
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