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核心结合因子急性髓性白血病中低甲基化剂单疗法:法国多中心回顾性研究

Ludovic Gabellier¹, Pierre Peterlin², Sylvain Thepot³

¹Département d'Hématologie Clinique, CHU Montpellier, Université Montpellier-Nîmes, 80, Avenue Augustin Fliche, 34090, Montpellier, France. l-gabellier@chu-montpellier.fr.

Annals of hematology

|January 25, 2024

概括

低甲基化剂 (HMA) 在核心结合因子急性髓性白血病 (CBF-AML) 中表现出适度的疗效,为复发/耐药患者或不适合强化化疗的患者提供了可容忍的选择.

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:

核心结合因子急性髓性白血病 (CBF-AML) 关于低甲基化剂 (HMA) 效率的数据有限.
HMA是一种用于治疗某些类型白血病的药物.

研究的目的:

评估HMA单一治疗在CBF-AML患者的特定亚组中的疗效和安全性.
评估前线和复发/耐药 (R/R) CBF-AML中的HMA治疗.

主要方法:

一项法国多中心回顾性研究包括49名患有CBF-AML的患者.
患者接受阿扎西蒂丁 (n=46) 或达西他 (n=3) 作为HMA单疗法.
收集了总体反应率 (ORR),总体存活率 (OS) 和不良事件的数据.

主要成果:

整体反应率 (ORR) 为49%,反应的中位时间为112天.
平均整体存活 (OS) 为10.6个月;响应者显著改善了OS (1年OS: 75%) 与非响应者 (1年OS: 15.3%).
在依赖输血或中性衰竭的患者中观察到血液学上的改善;不良事件与HMA安全概况一致.

结论:

HMA单疗显示中度临床活性,并且在R/RCBF-AML中耐受良好.
对于那些不能忍受强化化疗的CBF-AML患者来说,HMA是一种可行的治疗选择.

关键词:

急性骨髓性白血病 (AML) 是一种急性骨髓性白血病.亚扎西蒂丁是一种阿扎西蒂丁.核心约束因素是核心的约束因素.德西塔类药物德西塔类药物低甲基化剂是一种低甲基化剂.

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