Genomic characterization of paediatric acute lymphoblastic leukaemia: an opportunity for precision medicine therapeutics.
Genomic characterization of paediatric acute lymphoblastic leukaemia: an opportunity for precision medicine therapeutics.
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DOI：
10.1111/bjh.14474
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发表时间：
2017-03
影响因子：
6.5
通讯作者：
Hunger SP
中科院分区：
文献类型：
--
作者：
Tasian SK;Hunger SP
急性淋巴细胞白血病（ALL）的遗传和表观遗传特征分析的重大进展增强了对新发和复发ALL关键生物学亚群的理解，从而改善了患者的风险分层。这些成就进一步确定了关键的白血病相关途径和体细胞改变，这些途径和体细胞改变可能优先对激酶抑制剂、表观遗传疗法或其他新型药物治疗敏感。儿童ALL的治疗成功目前依赖于基于（1）基础生物学和临床特征和（2）初始治疗反应的深度以及适当调整化疗强度的患者的精细风险分层。本综述描述了儿童ALL的当前突变情况，并讨论了实施分子靶向治疗大幅改善生存率的机会。
Major advances in genetic and epigenetic profiling of acute lymphoblastic leukaemia (ALL) have enhanced the understanding of key biological subsets of de novo and relapsed ALL, which has led to improved risk stratification of patients. These achievements have further defined critical leukaemia-associated pathways and somatic alterations that may be preferentially sensitive to treatment with kinase inhibitors, epigenetic therapy or other novel agents. Therapeutic success in childhood ALL currently relies upon refined risk stratification of patients based on (1) underlying biological and clinical characteristics and (2) depth of initial treatment response with appropriate modulation of chemotherapy intensity. This review describes the current mutational landscape of childhood ALL and discusses opportunities for substantial improvements in survival with implementation of molecularly targeted therapies.