儿童急性髓系白血病靶向治疗研究进展

Research progress of targeted therapy of acute myeloid leukemia in children

急性髓系白血病(AML)占儿童白血病的15%~20%。尽管化疗和造血干细胞移植仍是儿童AML的一线治疗手段,但随着靶向药物的出现,极大程度上提高了AML的疗效,为患儿带来生存获益。靶向药物主要分为三类,第一类是突变位点抑制剂,如FLT3和IDH抑制剂;第二类是信号通路抑制剂,如Bcl-2抑制剂;第三类是表观遗传学药物,如DNA甲基化转移酶抑制剂和组蛋白去乙酰化酶抑制剂。本文就FLT3抑制剂、Bcl-2抑制剂及部分靶向药物的作用机制及临床研究进展进行综述,为儿童AML治疗选择的扩大化、精准化和个性化提供思路。

Acute myeloid leukemia(AML)accounts for 15%-20%of all childhood leukemia cases.Although chemotherapy and hematopoietic cell transplantation are still the first-line treatments for AML in children,the emergence of targeted drugs has greatly improved the efficacy of AML and brought survival benefits to children.Targeted drugs are mainly divided into three categories.The first type is mutant site inhibitors,such as FLT3 and IDH inhibitors.The second type is inhibitors of signaling pathway,such as Bcl-2 inhibitors.The third type is epigenetics drugs,such as DNA methyltransferase inhibitors and histone deacetylase inhibitors.This article describes the mechanisms of action and clinical research progress of FLT3 inhibitors,Bcl-2 inhibitors and some other targeted drugs to provide ideas for the expansion,precision and individuation of treatment selection for children with AML.