无进展生存-2的有效性和效率作为高级癌症随机试验中整体生存的替代终点
Rachel Woodford1, Deborah Zhou1, Peey-Sei Kok1
1NHMRC Clinical Trials Centre, University of Sydney, Camperdown, NSW, Australia.
JCO precision oncology
|January 11, 2024
概括
无进展生存期 (PFS) -2与总生存期 (OS) 的相关性比单独的PFS更强. 这种改善的代用终点只需要适度增加临床试验的随访时间和样本大小.
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:
- 无进展生存率 (PFS) 是癌症试验中常见的代用终点.
- 无进展生存期 (PFS) -2,定义为二线治疗进展的时间,可能为整体生存期 (OS) 提供更可靠的替代品.
- 与PFS相比,评估PFS-2的有效性和效率对于优化临床试验设计至关重要.
研究的目的:
- 为了比较PFS-2与PFS作为OS在先进实体瘤中的替代终点的有效性和效率.
- 评估与PFS和PFS-2的OS相对治疗效应之间的相关性.
- 确定与PFS相比,PFS-2的统计显著性和样本大小要求的时间.
主要方法:
- 进行了随机试验的电子搜索在先进的固体瘤中,以PFS,PFS-2和OS作为终点.
- 包括具有PFS-2测量和进展后随访定义协议的研究.
- 对比了与PFS和PFS-2的OS相对治疗效应的相关性,估计了统计学意义的时间和所需的人-年随访.
主要成果:
- 与PFS (r=0.46) 相比,PFS-2与OS (r=0.70) 的相关性更强.
- 具有PFS-2的OS (10.0个月) 和具有PFS-2的PFS (4.31个月) 与具有PFS的OS (16.59个月) 相比,获得统计学意义的时间更短.
- 与PFS相比,PFS-2需要额外156人年 (12.7%的增长) 的中位数才能达到统计学意义.
结论:
- PFS-2与整体存活率的相关性比PFS更好.
- 作为替代终点,PFS-2提供了更好的有效性,随着随访时间和样本大小的适度增加.
- 在未来的晚期癌症试验中,PFS-2应被视为主要终点.
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