Department of Pediatrics, College of Medicine, University of Cincinnati, Cincinnati, OH, USA.
Division of Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Paediatr Drugs. 2021 Sep;23(5):485-497. doi: 10.1007/s40272-021-00467-x. Epub 2021 Aug 22.
The outcomes associated with pediatric acute myeloid leukemia (AML) have improved over the last few decades, with the implementation of intensive chemotherapy, hematopoietic stem cell transplant, and improved supportive care. However, even with intensive therapy and the use of HSCT, both of which carry significant risks of short- and long-term side effects, approximately 30% of children are not able to be cured. The characterization of AML in pediatrics has evolved over time and it currently involves use of a variety of diagnostic tools, including flow cytometry and comprehensive genomic sequencing. Given the adverse effects of chemotherapy and the need for additional therapeutic options to improve outcomes in these patients, the genomic and molecular architecture is being utilized to inform selection of targeted therapies in pediatric AML. This review provides a summary of current, targeted therapy options in pediatric AML.
过去几十年中，通过强化化疗、造血干细胞移植和改善支持性护理，儿科急性髓系白血病（AML）的治疗效果得到了改善。然而，即使采用了高强度的治疗方法和造血干细胞移植，这两种方法都有显著的短期和长期副作用风险，但仍有约 30%的儿童无法治愈。儿科 AML 的特征随时间而演变，目前包括使用多种诊断工具，包括流式细胞术和全面基因组测序。鉴于化疗的不良反应以及需要额外的治疗选择来改善这些患者的预后，基因组和分子结构正在被用于为儿科 AML 中的靶向治疗选择提供信息。本文综述了儿科 AML 中目前的靶向治疗选择。
