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在不适合患者中新诊断的急性髓性白血病:2026年治疗算法

Naseema Gangat¹, Courtney D Dinardo²

¹Division of Hematology, Mayo Clinic, Rochester, MN, USA. gangat.naseema@mayo.edu.

Blood cancer journal

|August 16, 2025

概括

在不适合的患者中新诊断的急性髓性白血病 (ND-AML) 的管理现在标准使用venetoclax (Ven) 与低甲基化剂 (HMA). 结果因遗传学而异,向疗法为特定突变提供了选择.

科学领域:

血液学血液学血液学
在瘤学瘤学.
分子生物学分子生物学

背景情况:

新诊断的急性髓性白血病 (ND-AML) 的管理在不适合强化化疗的患者已经发展.
对AML疾病生物学的理解得到了改善,指导了新的治疗策略.
维持生活质量 (QoL) 是这一患者群体的一个关键目标.

研究的目的:

对不适合患者的ND-AML进行当前管理范式的审查.
讨论venetoclax (Ven) 和低甲基化剂 (HMA) 作为标准护理的作用.
探索基因突变对治疗反应和生存的影响.

主要方法:

对当前文献和临床试验数据的审查,对不适合患者的ND-AML管理.
基于遗传特征的治疗结果 (反应率,存活率) 的分析.
评估向疗法及其在组合疗法或单一疗法中的作用.

主要成果:

威尼托克拉克斯 (Ven) 加上低甲剂 (HMA) 是标准的护理,反应率 (CR/CRi) 为40-90%和可变的3年生存率 (0-67%).
瘤遗传学显著影响Ven-HMA结果; NPM1^MUT,IDH2^MUT,DDX41^MUT是有利的,而TP53^MUT,FLT3-ITD,K/NRAS^MUT是不利的.
同源干细胞移植 (ASCT) 对长期生存至关重要;三重组合和向疗法 (FLT3,IDH1/2,脑抑制剂) 提供了进一步的选择.

结论:

温-HMA疗法是有效的,但结果高度依赖于患者的遗传.
针对性疗法和三重组合显示出有前途,特别是在特定突变.
未来的研究应该专注于患者报告的结果,优化Ven-HMA疗程,并澄清ASCT时间.

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