Advances in targeted therapy for relapsed or refractory acute myeloid leukemia in children
熊润稷 1金润铭1
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作者信息
1. 华中科技大学同济医学院附属协和医院儿科,武汉 430022
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摘要
近年来,随着对儿童急性髓系白血病(acute myeloid leukemia,AML)遗传特征和分子标志物认识的深入,研究人员发现了与复发或难治性(relapsed or refractory,R/R)AML相关的潜在致病机制.这一发现促进了AML靶向治疗药物的开发与应用,并有望改善R/RAML患儿的预后和治疗效果.该文综述四类在儿童R/RAML的靶向治疗中取得进展的药物:靶向基因、靶向信号通路、靶向细胞表面抗原以及表观遗传抑制剂,旨在为临床医生和研究人员提供有益的参考和启示.
Abstract
In recent years,with the deepening understanding of the genetic characteristics and molecular markers of childhood acute myeloid leukemia(AML),researchers have identified potential pathogenic mechanisms related to relapsed or refractory(R/R)AML.This discovery has promoted the development and application of targeted therapeutic drugs for AML and is expected to improve the prognosis and therapeutic efficacy for children with R/R AML.This article reviews four classes of drugs that have made progress in the targeted therapy of R/R AML in children:gene-targeted therapies,signaling pathway targeting,targeting of cell surface antigens,and epigenetic inhibitors,with the aim of providing useful references and insights for clinicians and researchers.
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