Institute of Molecular Genetics and Genetic Engineering, Laboratory for Molecular Biomedicine, University of Belgrade, Serbia.
Institute of Molecular Genetics and Genetic Engineering, Laboratory for Molecular Biomedicine, University of Belgrade, Serbia.
Biochem Pharmacol. 2023 Sep;215:115705. doi: 10.1016/j.bcp.2023.115705. Epub 2023 Jul 31.
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.
急性髓细胞白血病（AML）是一种非常异质性的血液恶性肿瘤，约占所有儿科白血病病例的 20%。在过去几十年中，儿科 AML 的治疗效果有所改善，总生存率达到 70%左右。尽管如此，AML 仍然是导致儿童癌症高死亡率的主要类型之一。通过强化化疗、造血干细胞移植和优化支持性护理等标准治疗的调整，只能取得目前的效果，但要想显著改善儿科 AML 的治疗效果，就需要开发新的靶向治疗方法。近年来，基因组技术的进步极大地扩展了我们对 AML 生物学的认识，揭示了疾病的分子景观和复杂性，进而确定了新的治疗靶点。这篇综述简要概述了儿科 AML 的遗传景观，以及如何将其用于患者的精确分子特征分析和风险分层，以及开发有效的靶向治疗。此外，本文还介绍了分子靶向治疗和免疫治疗的最新进展，重点介绍了对儿科 AML 具有显著临床获益的治疗方法。
