癌症查随机临床试验的终点癌症阶段与死亡率相比:系统性审查和元分析
Xiaoshuang Feng1, Hana Zahed1, Justina Onwuka1
1Genomic Epidemiology Branch, International Agency for Research on Cancer, Lyon, France.
JAMA
|April 7, 2024
概括
第三到第四阶段的癌症发病率可以通过作为癌症特异性死亡率的替代终点来加速癌症查试验. 然而,它的适用性因癌症类型而异,影响多种癌症查测试的试验设计和解释.
科学领域:
- 癌症学
- 临床试验
- 生物统计学
背景情况:
- 随机临床试验 (RCT) 对于癌症查通常使用癌症特异性死亡率作为主要终点.
- 这种方法可以延长试验时间,延迟选试验结果的可用性.
- 第三至第四阶段的癌症发病率是一个潜在的替代终点,可以加快RCT的完成.
研究的目的:
- 将癌症特异性死亡率与癌症发病率作为癌症查RCT的终点的有效性进行比较.
- 评估不同癌症类型中癌症特异性死亡率和癌症发病率的降低之间的相关性.
主要方法:
- 进行了针对癌症查 (乳腺癌,结肠直肠癌,肺癌,卵巢癌,前列腺癌和其他癌症) 的41个RCT的元分析.
- 对干预和比较组的参与者数量,癌症诊断和死亡数据进行了提取.
- 使用皮尔森相关系数,线性回归和固定效应元分析来比较终点.
主要成果:
- 根据癌症类型,减少癌症特异性死亡率与III- IV癌症发病率之间的相关性有显著差异 (I2=65%,P=0. 02).
- 在卵巢 (ρ=0. 99) 和肺癌 (ρ=0. 92) 中观察到较高的相关性.
- 乳腺癌的相关性中等 (ρ=0. 70),而结直肠癌 (ρ=0. 39) 和前列腺癌 (ρ=- 0. 69) 的相关性较弱.
结论:
- 在某些癌症类型 (如卵巢和肺癌) 的查试验中,III-IV期癌症发病率可以作为癌症特异性死亡率的合适替代终点.
- 这一终点并不普遍适用,其实用性各不相同,需要在试验设计中仔细考虑,特别是在多种癌症查计划中.
- 这些发现对优化未来癌症查临床试验的设计和效率具有重要意义.
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