在癌症临床试验的第三阶段,对照组的过度表现
Ansel P Nalin1, Pavlos Msaouel2,3, Adam J Grippin1
1Department of Radiation Oncology, Division of Radiation Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Cancer
|February 7, 2026
概括
对照组的过度表现在癌症3期随机临床试验 (RCT) 中很常见,影响了研究功率. 当总生存率 (OS) 是主要终点时,这种现象更为普遍,这表明可能低估了后进展疗法.
科学领域:
- 瘤学 临床试验 临床试验
- 生物统计学 生物统计学
- 超流行病学 超流行病学
背景情况:
- 准确预测对照组的结果对于随机临床试验 (RCT) 的功率计算和入学目标至关重要.
- 对照组的意外过度表现可能导致研究不足,可能影响试验结果和结论.
研究的目的:
- 在第三阶段瘤学RCT中估计对照组超能力的患病率.
- 评估对照组过度表现和试验结果之间的关联.
主要方法:
- 在双臂,优势设计,第三阶段瘤学RCT上进行了一项元流行病学研究.
- 关于功率计算,理由和结果的数据是从出版物和协议中提取的.
- 对照组的表现被量化为观察到预试估计结果的比率;超强表现被定义为10%的改善.
主要成果:
- 在包括的385个RCT中,有43%的RCT表现出过高的性能.
- 过度表现与满足主要终点的几率较低有关 (aOR,0.989;P = 0.015).
- 使用整体存活率 (OS) 作为主要终点的RCT更有可能显示过度表现 (56%对33%;aOR,2.43;P=0.001).
结论:
- 对照臂的过度表现在3期瘤RCT中很常见,并影响研究功率.
- 在基于OS的试验中,表现过高的高患病率可能表明对后进展疗法的低估.
- 需要使用替代试验设计策略来更可靠地估计治疗效果.
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