对急性髓系白血病（AML）分子病理学的了解促进了治疗靶点的确定和相应新型靶向疗法的开发。自 2017 年以来，已有 12 种药物被批准用于治疗 AML 亚型：BCL2 抑制剂 Venetoclax； CD33抗体药物缀合物吉妥珠单抗奥佐米星；三种FLT3抑制剂（midostaurin、gilteritinib、quizartinib）；三种IDH抑制剂（针对IDH1突变的ivosidenib和olutasidenib；针对IDH2突变的enasidenib）；两种口服低甲基化药物（口服吸收性差的阿扎胞苷；完全吸收的地西他滨-西达氮啶[后者被批准作为骨髓增生异常综合征和慢性粒单核细胞白血病肠外低甲基化药物的替代药物，但常用于 AML]）； CPX-351（阿糖胞苷和柔红霉素摩尔比为 5:1 的封装脂质体）和 glasdegib（hedgehog 抑制剂）。其他靶向治疗（menin 抑制剂、CD123 抗体药物偶联物）也显示出有希望的结果。为了在像 AML 这样罕见且异质的实体中获得最佳结果，需要专业知识、对这种罕见癌症的熟悉，以及在严格的支持性护理条件下获得和提供不同的治疗方法。在这篇综述中，我们更新了护理标准和研究疗法，并概述了当前和未来有希望的研究方向。© 2024。作者。

The understanding of the molecular pathobiology of acute myeloid leukemia (AML) has spurred the identification of therapeutic targets and the development of corresponding novel targeted therapies. Since 2017, twelve agents have been approved for the treatment of AML subsets: the BCL2 inhibitor venetoclax; the CD33 antibody drug conjugate gemtuzumab ozogamicin; three FLT3 inhibitors (midostaurin, gilteritinib, quizartinib); three IDH inhibitors (ivosidenib and olutasidenib targeting IDH1 mutations; enasidenib targeting IDH2 mutations); two oral hypomethylating agents (oral poorly absorbable azacitidine; fully absorbable decitabine-cedazuridine [latter approved as an alternative to parenteral hypomethylating agents in myelodysplastic syndrome and chronic myelomonocytic leukemia but commonly used in AML]); and CPX-351 (encapsulated liposomal 5:1 molar ratio of cytarabine and daunorubicin), and glasdegib (hedgehog inhibitor). Other targeted therapies (menin inhibitors, CD123 antibody-drug conjugates) are showing promising results. To achieve optimal results in such a rare and heterogeneous entity as AML requires expertise, familiarity with this rare cancer, and the access to, and delivery of disparate therapies under rigorous supportive care conditions. In this review, we update the standard-of-care and investigational therapies and outline promising current and future research directions.© 2024. The Author(s).