文章：

急性髓系白血病分化疗法的最新进展

Recent Developments in Differentiation Therapy of Acute Myeloid Leukemia

原文发布日期：28 March 2025

DOI: 10.3390/cancers17071141

类型: Article

开放获取: 是

英文摘要：

Acute myeloid leukemia (AML) is characterized by the clonal expansion of myeloid progenitors blocked at various stages of their differentiation process, and drugs that bypass this differentiation block are therapeutically efficient, as shown by retinoic acid and arsenic trioxide in acute promyelocytic leukemia. However, the successful application of differentiation therapy in APL has not translated into clinical benefit for other non-APL subtypes of AML, in which intensive chemotherapy regimens represent the standard of care. However, the development of molecular studies has led to the identification of therapeutic targets (such as mutated proteins and deregulated pathways) and has led to the generation of a new category of specific pharmacologic agents. Some of these agents, such as inhibitors of mutant isocitrate dehydrogenase (IDH1 and IDH2), lysine-specific demethylase-1 (LSD1), and Menin, have shown the capacity to induce leukemic cell differentiation and with significant therapeutic efficacy.

摘要翻译： 

急性髓系白血病（AML）的特征在于髓系祖细胞在分化过程的不同阶段受阻并发生克隆性扩增，能够绕过这种分化阻滞的药物具有治疗效力，这在急性早幼粒细胞白血病中通过维甲酸和三氧化二砷的应用已得到证实。然而，分化疗法在APL中的成功应用并未转化为对其他非APL亚型AML的临床获益，对于这些亚型，强化化疗方案仍是标准治疗方案。不过，分子研究的发展促进了对治疗靶点（如突变蛋白和失调通路）的识别，并催生了一类新型特异性药物。其中部分药物，如突变型异柠檬酸脱氢酶（IDH1和IDH2）抑制剂、赖氨酸特异性去甲基化酶-1（LSD1）抑制剂和Menin抑制剂，已展现出诱导白血病细胞分化并产生显著治疗效果的能力。

原文链接：

Recent Developments in Differentiation Therapy of Acute Myeloid Leukemia