两阶段随机临床试验与右审查的终点:频率主义和贝叶斯适应性设计的比较
Luana Boumendil1,2,3, Sylvie Chevret1,2,3, Vincent Lévy1,4,5
1INSERM U1153, Team ECSTRRA, Hôpital Saint Louis, Paris, France.
Statistics in medicine
|June 6, 2024
概括
具有时间到事件结果的自适应性临床试验需要强大的分析方法. 对于具有样本大小调整的适应性设计,建议采用频率主义方法,特别是Wassmer和Jörgens的方法,特别是在功能不足的试验中.
科学领域:
- 生物统计学 生物统计学
- 临床试验设计 临床试验设计
- 生存分析的分析.
背景情况:
- 适应性随机临床试验对于长时间的时间到事件结果至关重要.
- 对于定义停止边界和将统计数据结合到频率主义适应设计中,缺乏共识.
研究的目的:
- 将适应性临床试验的频率主义和贝叶斯主义方法与时间到事件结果进行比较.
- 评估单面频率主义设计中样本大小调整和终端分析的方法.
主要方法:
- 模拟研究将三个频率主义方法 (Wassmer,Desseaux,Jörgens) 与贝叶斯式监测方法 (Freedman) 的比较.
- 设计包括一个单一的中间分析,有效性停止规则和潜在的样本大小调整.
- 评估重点是I型错误率控制和统计能力.
主要成果:
- 所有测试的方法都控制了I型错误率,除了贝叶斯方法.
- 频率主义方法表现出令人满意的力量,在功能不足的场景中表现优于贝叶斯方法.
- 相对危险假设对所有方法的功率产生了重大影响.
结论:
- 频率主义方法,特别是Wassmer和Jörgens,对于具有生存终点和样本大小适应的适应性设计是首选的,特别是在可能违反比例危险假设的情况下.
- 与贝叶斯式监测相比,这些方法在不足的试验中提供了更好的性能.
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