评估瘤临床试验的早期干预:系统性审查
Apoorva Kakkilaya1, Aaron Trando2, Edward R Scheffer Cliff3
1John Sealy School of Medicine, University of Texas Medical Branch, Galveston, TX, United States.
The oncologist
|September 5, 2024
概括
早期治疗燃烧的多发性骨髓瘤 (SMM) 没有明显的好处比治疗活跃的多发性骨髓瘤 (MM). 一项审查发现,没有令人信服的证据表明,早期干预SMM导致更好的结果,与MM的晚期治疗相比.
科学领域:
- 血液学 血液学 血液学
- 临床试验 临床试验
- 骨髓瘤研究 骨髓瘤研究
背景情况:
- 燃烧性多发性骨髓瘤 (SMM) 是一个无症状的阶段,在活跃的多发性骨髓瘤 (MM) 之前.
- 从SMM发展为MM的风险各不相同,对最佳治疗时间和疗效的共识有限.
- 现有的研究缺乏明确的理解,即SMM的早期干预是否能改善患者的治疗结果.
研究的目的:
- 系统地审查所有潜在的SMM干预临床试验.
- 将SMM中使用的治疗方案的疗效与新诊断的多发性骨髓瘤 (NDMM) 中的疗效进行比较.
- 为了确定早期的SMM治疗是否导致与NDMM相比更深或更有效的反应.
主要方法:
- 在2023年4月1日之前对SMM进行前性干预临床试验进行了系统审查.
- 包括已发表的研究,会议摘要,以及来自ClinicalTrials.gov.gov的未发表的试验.
- 捕获的试验变量包括治疗策略和疗效,重点关注整体存活率 (OS) 和生活质量.
主要成果:
- 确定了45个SMM试验;38个使用活性髓瘤药物,7个使用骨修饰剂.
- 在18项随机试验中,只有1项专注于OS;无进展生存率是最常见的初级终点.
- 在SMM和NDMM两种疗法中测试的6种疗法中,其中5种疗法的SMM反应率较低 (例如32% vs. 63% ≥VGPR).
结论:
- 在SMM试验设计中存在显著的变化,包括终点和干预措施.
- 尽管有统计上的局限性,但对比方案并没有显示出令人信服的证据表明,SMM的早期治疗比NDMM更有效.
- 需要进一步的研究来确定SMM的最佳治疗策略.
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