儿童急性髓细胞白血病基因突变与靶向药物治疗的研究进展  

Research Advance of Gene Mutation and Targeted Drug Therapy in Childhood Acute Myeloid Leukemia——Review

作　　者：杜成坎 卢莹 DU Cheng-Kan;LU Ying(Department of Pathophysiology,Shanghai Jiaotong University School of Medicine,Shanghai 200025,China)

机构地区：[1]上海交通大学医学院基础医学院病理生理学系,上海200025

出　　处：《中国实验血液学杂志》2022年第2期631-635,共5页Journal of Experimental Hematology

摘　　要：急性髓细胞白血病(AML)的临床治疗方案在儿童与成人中并无显著区别,均采用去甲氧柔红霉素和阿糖胞苷组成的IA为基础的方案诱导化疗。随着测序技术的飞速发展,人们对AML分子生物学异常的了解不断加深,各类基于AML基因突变的靶向药物的开发和应用进展迅速。本文对AML中几种常见的基因突变(FLT3、NPM1、C/EBPA等)进行描述,并将已用于临床治疗或已报道的靶向治疗药物(酪氨酸激酶抑制剂、IDH1突变抑制剂以及表观遗传修饰抑制剂)在儿童及成人AML患者中的疗效以及差异进行总结。The clinical therapeutic regimen for acute myeloid leukemia(AML) is not significantly different between adults and children, which is mostly based on IA(idarubicin and cytosine arabinoside) induction chemotherapy. With the rapid development of sequencing technique, people′s understandings towards the molecular and biological abnormalities of AML are increasing, diverse AML gene mutation-based targeted drugs have been rapidly developed and applied. In this review, several commonly gene mutations in AML(such as FLT3, NPM1 and C/EBPA) was described, and the therapeutic effects and differences of targeted drugs that used in clinical treatment or had been reported(like tyrosine kinase inhibitor, IDH1 mutation inhibitor and epigenetic modification inhibitor) in child and adult AML patients were summrized.

关 键 词：急性髓细胞白血病 基因突变 靶向治疗 儿童 

分 类 号：R733.71[医药卫生—肿瘤]