At present, acute myeloid leukemia (AML) accounts for about 15%-20% of childhood acute leukemia. Although overall survival rate is increasing with the help of risk stratification, stratification of chemotherapy, and supportive treatment, conventional pharmacotherapy still has a limited clinical effect and certain limitations in improving remission rate in previously untreated patients and reducing recurrence after remission. With the development of precision medicine, the mechanisms of targeted therapy, including abnormal activation of AML-related signaling pathways and epigenetic modification, have been found in recent years. Molecular-targeted drugs can therefore act on specific receptors and target genes to improve clinical effect and the prognosis of AML patients.
急性髓系白血病（AML）约占儿童急性白血病的15%~20%，虽然在危险度分级、分层化疗以及支持治疗等手段下AML的总体生存率较前升高，但是传统治疗下的临床疗效仍然有限，且在提高初治缓解率及减少缓解后复发方面存在局限性。近年来，随着精准医疗的不断发展，靶向治疗机制即包括AML相关信号通路的异常激活以及表观遗传修饰等研究不断深入，分子靶向药物可针对于特定的受体及目的基因等发挥作用，从而增加疗效和改善AML患者预后。