在第三阶段瘤学试验中,生存终点的预期与观察效应大小
Sophia J Lamp1,2, Gina L Mazza2
1Department of Psychology, Arizona State University, Tempe, AZ, USA.
Journal of the National Cancer Institute
|July 2, 2025
概括
第三阶段瘤学试验经常由于过度估计的效果大小而失败. 这一系统性审查发现生存终点估计过高,表明试验可能不足,从而增加了癌症研究的失败率.
科学领域:
- 在瘤学瘤学.
- 临床试验设计 临床试验设计
- 生物统计学 生物统计学
背景情况:
- 随机的3期瘤学试验经常失败的主要终点.
- 第一阶段和第二阶段的试验结果并不总是预测第三阶段的成功.
- 对试验功率的效果大小估计是一个关键因素.
研究的目的:
- 调查在3期瘤学试验中的效果大小选择实践.
- 为了确定是否对生存终点的影响大小被高估.
- 评估这些做法对试验功率和失败率的影响.
主要方法:
- 在2023年发表的第三阶段瘤学试验的系统审查.
- 对生存终点的影响大小的分析,特别是危险比率 (HR).
- 用于功率计算的预期HRs与初级分析中观察到的HRs的比较.
主要成果:
- 确定了对生存终点的效果大小估计过高的模式.
- 预期危险比率 (平均HR = 0.66) 强于观察到的 (平均HR = 0.72).
- 在111项试验中,57.3%的观察到的危险比率低于预期.
结论:
- 由于对效果大小的高估,第三期瘤学试验可能会被系统地低估.
- 低能量的试验对瘤学研究的高失败率有很大影响.
- 修订效果大小选择实践对于改善瘤学临床试验的成功至关重要.
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