佩米加替尼治疗肌肉/淋巴瘤新生体与FGFR1重组
Srdan Verstovsek1, Jean-Jacques Kiladjian2, Alessandro M Vannucchi3
1University of Texas MD Anderson Cancer Center, Houston.
在治疗有纤维细胞生长因子受体1重组 (MLN- FGFR1) 的骨髓/ 淋巴瘤中,特别是慢性期患者中,佩米加替尼具有很高的疗效. 经过剂量调整,治疗是可以管理的,这表明佩米加替尼
科学领域:
- 血液学
- 癌症学
- 分子生物学
背景情况:
- 带有纤维细胞生长因子受体1重组 (MLN- FGFR1) 的骨髓状/ 淋巴状瘤是侵袭性的血液恶性瘤.
- 这些瘤是由由染色体8p11重组引起的构成性FGFR1激活引起的.
- 目前对MLN-FGFR1的治疗选择有限,突出显示需要新的治疗策略.
研究的目的:
- 评估FGFR1-3抑制剂佩米加替尼在MLN-FGFR1患者中的疗效和安全性.
- 评估佩米加替尼的反应率,包括完整的反应和完整的细胞遗传反应.
- 根据疾病阶段 (慢性与爆发阶段) 分析治疗结果.
主要方法:
- 一项2期单臂研究 (FIGHT-203) 招募了确诊的MLN- FGFR1患者.
- 患者接受口服佩米加替尼13. 5毫克,每天服用一次,每周休息2周或连续服用.
- 主要终点是完整反应率;次要终点包括完整细胞遗传反应率和安全性.
主要成果:
- 总体完整反应率为74% (31/ 42),慢性期患者为96%;爆发期患者为44%.
- 73% (33/45) 的患者获得了完整的细胞遗传反应.
- 超血 (76%) 是最常见的不良事件;口腔炎 (19%) 是最常见的3+级事件.
结论:
- 在慢性阶段的MLN- FGFR1中,佩米加替尼显示出显著的疗效,几乎达到完全的反应.
- 这种药物在爆发期患者中表现出显著的反应率,表明其具有治疗潜力.
- 经过剂量调整,佩米加提尼布在一般情况下是可控的,这表明了良好的安全性.
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