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交叉验证的风险评分适应性丰富 (CADEN) 设计
Svetlana Cherlin1, James M S Wason1
1Population Health Sciences Institute, Newcastle University, Baddiley-Clark Building, Newcastle upon Tyne, UK.
Contemporary clinical trials
|July 8, 2024
概括
本研究介绍了用于临床试验的交叉验证的适应性丰富 (CADEN) 设计. 通过丰富预测对治疗反应最好的患者的试验,CADEN提高了统计效率和患者的益处.
科学领域:
- 临床试验的设计
- 生物统计学 生物统计学
- 翻译医学是一种翻译医学.
背景情况:
- 传统的临床试验设计可能无法最佳地确定从治疗中获益最多的患者子组.
- 丰富策略旨在通过专注于潜在的响应群体来提高试验效率.
研究的目的:
- 提出和评估一种新的适应性丰富试验设计,交叉验证的适应性丰富 (CADEN).
- 使用模拟研究,将CADEN的性能与非丰富设计 (CVRS) 的性能进行比较.
- 在实际的临床试验环境中展示CADEN的实际应用.
主要方法:
- 在CADEN设计中,使用从基线患者数据中得出的风险评分来丰富试验群体的预测响应者.
- 它包含了一个早期停止无用的规则.
- 通过对CADEN与交叉验证风险得分 (CVRS) 设计进行比较的模拟来评估性能.
主要成果:
- 在存在敏感患者亚组时,CADEN 显示了更高的统计能力.
- 与CVRS设计相比,设计导致预期样本大小减少.
- 在两个真实临床试验中展示了CADEN的应用.
结论:
- 与现有的非富化方法相比,CADEN提供了更好的统计效率和更大的患者益处.
- 拟议的设计提高了检测响应子组治疗效应的能力.
- 通过R包提供CADEN方法论,以便更广泛地实施.
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