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.
Advances in targeted therapy for relapsed or refractory acute myeloid leukemia in children
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.
万方数据
