作为前线治疗慢性骨髓性白血病在慢性阶段ponatinib的结果
Fadi G Haddad1, Koji Sasaki1, Lewis Nasr1
1Department of Leukemia, The University of Texas M.D. Anderson Cancer Center, Houston, Texas, USA.
Cancer
|May 28, 2024
概括
波纳替尼在慢性髓性白血病中表现出高的完全细胞遗传反应 (CCyR) 和分子缓解率. 然而,心血管毒性限制了其在前线环境中的长期使用.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 波纳替尼是第三代BCR::ABL1氨酸激酶抑制剂 (TKI),在费城染色体阳性白血病中已证明有效.
- 这项研究报告了慢性阶段慢性髓性白血病中波纳替尼二期试验的长期随访数据.
研究的目的:
- 评估ponatinib在慢性阶段慢性髓性白血病患者的长期疗效和安全性.
- 评估细胞遗传和分子反应率,无事件存活率和整体存活率.
主要方法:
- 患者接受了ponatinib,剂量在30至45毫克/天之间.
- 主要终点是6个月完整细胞遗传反应 (CCyR) 的速度.
- 由于心血管毒性问题,该试验被停止,这促使许多患者的TKI变化.
主要成果:
- 6个月后观察到高CCyR (96%),主要分子响应 (78%),MR4 (50%) 和MR4.5 (36%) 的高率.
- 67%的患者在24个月和51%的患者在60个月内实现了持久的分子缓解 (MR4).
- 10年整体存活率为90%,其中16%的人经历了严重的心血管不良事件.
结论:
- 在新诊断的慢性髓性白血病中,ponatinib 取得了显著的细胞遗传和分子反应.
- 动脉/血管封闭性和其他严重的毒性限制了波纳替尼作为一线治疗的使用.
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