急性髓系白血病（AML）是一种非常异质的血液系统恶性肿瘤，约占所有儿童白血病病例的20％。过去几十年来，儿童AML的预后已经有所改善，总体生存率提高至70％。尽管如此，由于其高死亡率，AML仍然是儿童癌症中的主要类型之一。标准治疗方法的调节，如化疗强化，造血干细胞移植和优化的支持性护理，只能达到目前的程度，但为了在儿童AML的结果方面取得重大改善，必须发展新的靶向治疗方法。近年来，基因组技术的进展极大地扩展了我们对AML生物学的认识，揭示了该病的分子景观和复杂性，这反过来导致了新的治疗靶点的发现。本综述简要概述了儿童AML的遗传景观，并介绍了其用于对患者进行精确分子特征和风险分层以及有效靶向治疗策略的开发。此外，本综述重点介绍了分子靶向治疗和免疫治疗方面的最新进展，强调对儿童AML具有显著临床益处的治疗方法。版权所有© 2023 Elsevier Inc. All rights reserved.

Acute myeloid leukemia (AML) is a very heterogeneous hematological malignancy that accounts for approximately 20% of all pediatric leukemia cases. The outcome of pediatric AML has improved over the last decades, with overall survival rates reaching up to 70%. Still, AML is among the leading types of pediatric cancers by its high mortality rate. Modulation of standard therapy, like chemotherapy intensification, hematopoietic stem cell transplantation and optimized supportive care, could only get this far, but for the significant improvement of the outcome in pediatric AML, development of novel targeted therapy approaches is necessary. In recent years the advances in genomic techniques have greatly expanded our knowledge of the AML biology, revealing molecular landscape and complexity of the disease, which in turn have led to the identification of novel therapeutic targets. This review provides a brief overview of the genetic landscape of pediatric AML, and how it's used for precise molecular characterization and risk stratification of the patients, and also for the development of effective targeted therapy. Furthermore, this review presents recent advances in molecular targeted therapy and immunotherapy with an emphasis on the therapeutic approaches with significant clinical benefits for pediatric AML.Copyright © 2023 Elsevier Inc. All rights reserved.