是时候抵制使用RECIST作为罕见瘤试验的主要终点了吗?
Brian A Van Tine1,2,3, Sasha Haarberg1,2
1Division of Medical Oncology, Department of Medicine, School of Medicine, Washington University in St. Louis, St. Louis, Missouri.
概括
根据RECIST标准,用特拉美丁尼布治疗表皮性血管内瘤 (EHE) 没有显示瘤缩. 然而,患者报告说疼痛明显改善,这表明生活质量有所改善.
科学领域:
- 在瘤学瘤学.
- 癌症遗传学 癌症遗传学
- 分子向疗法分子向疗法
背景情况:
- 皮质状血管内皮瘤 (EHE) 是一种极为罕见的血管瘤.
- YAP-CAMTA1基因融合是EHE的主要驱动力.
- 众所周知,YAP-CAMTA1融合激活了MEK信号通路.
研究的目的:
- 评估Trametinib,一种MEK抑制剂,在患有EHE的患者中的疗效.
- 用RECIST标准和患者报告的结果来评估瘤反应.
主要方法:
- 进行了第二阶段临床试验 (SARC33).
- 患有EHE的患者接受了特拉美丁尼布.
- 用RECIST标准评估瘤的反应.
- 收集了患者报告的结果,包括疼痛.
主要成果:
- 该试验没有达到基于RECIST标准的首要目标.
- 特拉美替尼布在患者报告的结果中显示出显著的益处,特别是改善了疼痛.
- 通过RECIST.没有观察到任何客观的瘤缩.
结论:
- 特拉美提尼布虽然不能在EHE中实现RECIST定义的瘤减少,但在症状管理方面具有潜在的好处.
- 患者报告的结果表明,MEK抑制在改善EHE患者的生活质量方面发挥了作用.
- 进一步调查特拉美丁尼布在EHE症状控制中的作用是合理的.
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