无进展生存率作为复发性或耐药性多发性髓瘤患者的整体生存率的替代终点
Meletios Dimopoulos1, Pieter Sonneveld2, Salomon Manier3
1Department of Clinical Therapeutics, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece.
BMC cancer
|April 29, 2024
概括
无进展生存率 (PFS) 强烈预测复发性/耐药性多发性骨髓瘤 (RRMM) 的整体生存率 (OS). 每一个1个月的PFS增加与1.72个月的OS增加相关,支持PFS作为RRMM临床试验中可靠的终点.
科学领域:
- 血液学 血液学 血液学
- 临床试验 临床试验
- 生物统计学 生物统计学
背景情况:
- 复发性/耐药性多发性骨髓瘤 (RRMM) 带来了重大的治疗挑战.
- 无进展生存率 (PFS) 和总生存率 (OS) 是评估RRMM疗法的关键终点.
- 了解PFS和OS之间的定量关系对于试验设计和解释至关重要.
研究的目的:
- 量化评估RRMM患者中PFS中位数和OS中位数之间的关系.
- 根据已发表的随机对照试验 (RCT) 来评估这种关系.
- 确定PFS是否可以可靠地预测RRMM中的OS.
主要方法:
- 在 Embase,PubMed,MEDLINE 和 EBM 评论 (1970-2022) 的系统文献搜索.
- 包括31个RCT,包括56个治疗臂和10,450名RRMM患者.
- 使用斯皮尔曼和皮尔森相关系数和加权最小平方回归的统计分析.
主要成果:
- 在中位数PFS和中位数OS之间观察到强烈的正相关性 (斯皮尔曼r=0.80,皮尔森r=0.79,P<0.0001).
- 每增加1个月的中位数PFS与1.72个月的中位数OS (95% CI 1.26-2.17) 的增加有关.
- 平均PFS中位数为7.1个月,平均OS中位数为28.1个月.
结论:
- 这些发现证实了使用PFS作为RRMM中OS的替代终点.
- 即使包括最近的临床试验数据,这种关系也是如此.
- 在RRMM中,PFS是治疗疗效的有价值的预测标志物.
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