The first 5 decades of research in acute myeloid leukemia (AML) were dominated by the cytarabine plus anthracyclines backbone, with advances in strategies including allogeneic hematopoietic stem cell transplantation, high-dose cytarabine, supportive care measures, and targeted therapies for the subset of patients with acute promyelocytic leukemia. Since 2017, a turning point in AML research, 12 agents have received regulatory approval for AML in the United States: venetoclax (BCL2 inhibitor); gemtuzumab ozogamicin (CD33 antibody–drug conjugate); midostaurin, gilteritinib, and quizartinib (fms-like tyrosine kinase 3 inhibitors); ivosidenib, olutasidenib, and enasidenib (isocitrate dehydrogenase 1 and 2 inhibitors); oral azacitidine (a partially absorbable formulation); CPX351 (liposomal encapsulation of cytarabine:daunorubicin at a molar ratio of 5:1); glasdegib (hedgehog inhibitor); and recently revumenib (menin inhibitor; approved November 2024). Oral decitabine-cedazuridine, which is approved as a bioequivalent alternative to parenteral hypomethylating agents in myelodysplastic syndrome, can be used for the same purpose in AML. Menin inhibitors, CD123 antibody–drug conjugates, and other antibodies targeting CD123, CD33, and other surface markers are showing promising results. Herein, the authors review the frontline and later line therapies in AML and discuss important research directions.

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2025 年急性髓系白血病的管理和研究


急性髓系白血病 （AML） 的前 5 年研究以阿糖胞苷加蒽环类药物为主，策略的进展包括同种异体造血干细胞移植、大剂量阿糖胞苷、支持性护理措施和针对急性早幼粒细胞白血病患者亚群的靶向治疗。自 2017 年（AML 研究的转折点）以来，美国已有 12 种药物获得 AML 监管批准：venetoclax（BCL2 抑制剂）;吉妥珠单抗奥佐米星（CD33 抗体 - 药物偶联物）;米哚妥林、吉替替尼和 quizartinib（FMS 样酪氨酸激酶 3 抑制剂）;Ivosidenib、olutasidenib 和 enasidenib（异柠檬酸脱氢酶 1 和 2 抑制剂）;口服阿扎胞苷（一种部分可吸收的制剂）;CPX351 （阿糖胞苷：柔红霉素的脂质体包膜，摩尔比为 5：1）;glasdegib（刺猬抑制剂）;以及最近的 revumenib（menin 抑制剂;2024 年 11 月批准）。口服地西他滨-西达哌啶被批准作为骨髓增生异常综合征肠外低甲基化药物的生物等效替代品，可用于 AML 的相同目的。Menin 抑制剂、CD123 抗体-药物偶联物和其他靶向 CD123、CD33 和其他表面标志物的抗体显示出有希望的结果。本文综述了 AML 的一线和后续一线治疗，并讨论了重要的研究方向。