Goyama Susumu
Division of Cellular Therapy, the Institute of Medical Science, the University of Tokyo.
Rinsho Ketsueki. 2016 Feb;57(2):118-28. doi: 10.11406/rinketsu.57.118.
Acute myeloid leukemia (AML) is a form of blood cancer that is characterized by the rapid growth of abnormal myeloid cells. Although the general therapeutic strategy in patients with AML has not changed substantially in more than 30 years, remarkable progress has been achieved in understanding the pathogenesis of AML. Genome-wide analyses have revealed genetic mutations and epigenetic dysregulations that are present in AML cells. Studies of leukemia stem cells have clarified their complex properties and functions in the development of AML, and have also led to the recent identification of pre-leukemic hematopoietic stem cells that undergo clonal evolution in healthy people. Translation of these new findings into the clinical setting is just beginning. This article focuses on recent advances in basic research on the molecular pathogenesis of AML. New strategies under investigation, including epigenetic therapies and immunotherapies, to provide better therapeutic options for AML patients, are also summarized.
急性髓系白血病（AML）是一种血癌，其特征是异常髓系细胞快速生长。尽管30多年来AML患者的总体治疗策略没有实质性改变，但在理解AML发病机制方面已取得显著进展。全基因组分析揭示了AML细胞中存在的基因突变和表观遗传失调。对白血病干细胞的研究阐明了它们在AML发生发展中的复杂特性和功能，还导致最近发现了在健康人中经历克隆进化的白血病前期造血干细胞。将这些新发现转化为临床应用才刚刚开始。本文重点关注AML分子发病机制基础研究的最新进展。还总结了正在研究的新策略，包括表观遗传疗法和免疫疗法，以为AML患者提供更好的治疗选择。
