急性髓系白血病 (AML) 是一种克隆性造血系统癌症，会破坏正常的造血功能，最终导致骨髓衰竭和死亡。在美国，AML 的年发病率为每 10 万人 4.1 例，65 岁以上患者的发病率更高。急性髓系白血病包括许多具有异质分子特征、治疗反应和预后的亚组。本综述讨论了支持 AML 一线治疗的证据、指导治疗的主要原则以及分子靶向治疗的进展。急性髓系白血病是一种遗传复杂的动态疾病。最常见改变的基因包括 FLT3、NPM1、DNMT3A、IDH1、IDH2、TET2、RUNX1、NRAS 和 TP53。这些改变的发生率因患者年龄、既往血液癌症病史以及之前接受过任何癌症的化疗和/或放疗而异。自2010年以来，分子数据已被纳入AML预测，逐渐导致靶向治疗纳入诱导化疗的初始治疗方法和后续管理。第一个分子靶向抑制剂米斯托林 (midostaurin) 于 2017 年被批准用于治疗携带 FLT3 变异的 AML 患者。从那时起，人们对 AML 分子发病机制的了解不断扩大，从而能够确定药物治疗的其他潜在靶点，将分子治疗纳入治疗中。靶向治疗（针对FLT3变异体的米斯托林、吉尔特替尼和奎扎替尼；针对IDH1变异体的ivosidenib和olutasidenib，以及针对IDH2变异体的enasidenib），以及合理的联合治疗方案的确定。低甲基化药物与维奈托克联合用药的批准彻底改变了老年人 AML 的治疗，比单一疗法延长了生存期。此外，现在患者转诊进行造血细胞移植的依据更加合理。在基因组医学时代，AML治疗是根据患者的合并症和AML基因组谱定制的。

Acute myeloid leukemia (AML) is a clonal hematopoietic cancer that disrupts normal hematopoiesis, ultimately leading to bone marrow failure and death. The annual incidence rate of AML is 4.1 per 100 000 people in the US and is higher in patients older than 65 years. Acute myeloid leukemia includes numerous subgroups with heterogeneous molecular profiles, treatment response, and prognosis. This review discusses the evidence supporting frontline therapies in AML, the major principles that guide therapy, and progress with molecularly targeted therapy.Acute myeloid leukemia is a genetically complex, dynamic disease. The most commonly altered genes include FLT3, NPM1, DNMT3A, IDH1, IDH2, TET2, RUNX1, NRAS, and TP53. The incidence of these alterations varies by patient age, history of antecedent hematologic cancer, and previous exposure to chemotherapy and/or radiotherapy for any cancer. Since 2010, molecular data have been incorporated into AML prognostication, gradually leading to incorporation of targeted therapies into the initial treatment approach of induction chemotherapy and subsequent management. The first molecularly targeted inhibitor, midostaurin, was approved to treat patients with AML with FLT3 variants in 2017. Since then, the understanding of the molecular pathogenesis of AML has expanded, allowing the identification of additional potential targets for drug therapy, treatment incorporation of molecularly targeted therapies (midostaurin, gilteritinib, and quizartinib targeting FLT3 variants; ivosidenib and olutasidenib targeting IDH1 variants, and enasidenib targeting IDH2), and identification of rational combination regimens. The approval of hypomethylating agents combined with venetoclax has revolutionized the therapy of AML in older adults, extending survival over monotherapy. Additionally, patients are now referred for hematopoietic cell transplant on a more rational basis.In the era of genomic medicine, AML treatment is customized to the patient's comorbidities and AML genomic profile.