儿童白血病诊治研究——精准医学实践典范  

Research in the diagnosis and treatment of childhood leukemia:A model in practising precision medicine WANG

作　　者：王天有[1] 李志刚[1] 

机构地区：[1]首都医科大学附属北京儿童医院,北京100045

出　　处：《中国实用儿科杂志》2016年第4期241-245,共5页Chinese Journal of Practical Pediatrics

摘　　要：儿童白血病的疗效已取得极大提高。近年来,大量新的分子遗传学异常被发现,使得临床分型更为准确,应用高通量测序技术可识别出极低水平的微小残留病,小分子靶向药物、单克隆抗体治疗、嵌合型抗原受体修饰等新型治疗技术的应用,给攻克难治、复发白血病带来新的希望,以6-巯基嘌呤代谢相关基因多态性为代表的药物遗传学研究为在提高疗效的同时降低毒副反应做出了突出贡献。这些进步无不深刻体现着精准医学的理念与精神,并为今后的研究奠定坚实的基础。Great progress has been made in treatment outcome of children with leukemia. Recently,clinical classification has been more accurate,resulting from discovery of many molecular genetic anomalies. High throughput sequencing has made it possible to identify minimal residual disease at very low level. New therapy approaches,such as small molecule targeted drugs,monoclonal antibodies,and chimeric antigen receptor modification,bring hope to the treatment of refractory/relapsed leukemia. Pharmacogenetics research,represented by the polymorphisms of 6-mercaptopurine metabolism related genes,has made great contribution to the toxicity reduction. The progress deeply reflects the concepts and essence of precision medicine. Furthermore,the results lay a solid foundation for future research.

关 键 词：急性淋巴细胞白血病 急性髓细胞白血病 微小残留病 无事件生存率 

分 类 号：R72[医药卫生—儿科]