Pan Darren, Rampal Raajit, Mascarenhas John
Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY.
Leukemia Service, Memorial Sloan Kettering Cancer Center, New York, NY; and.
Blood Adv. 2020 Mar 10;4(5):970-982. doi: 10.1182/bloodadvances.2019001245.
Acute myeloid leukemia (AML) is a highly heterogeneous disease arising from acquired genetic and epigenetic aberrations which stifle normal development and differentiation of hematopoietic precursors. Despite the complex and varied biological underpinnings, induction therapy for AML has remained fairly uniform over 4 decades and outcomes remain poor for most patients. Recently, enhanced understanding of the leukemic epigenome has resulted in the translational investigation of a number of epigenetic modifying agents currently in various stages of clinical development. These novel therapies are based on mechanistic rationale and offer the potential to improve AML patient outcomes. In light of many recent advances in this field, we provide an updated, clinically oriented review of the evolving landscape of epigenetic modifying agents for the treatment of AML.
急性髓系白血病（AML）是一种高度异质性疾病，由获得性遗传和表观遗传畸变引起，这些畸变会抑制造血前体细胞的正常发育和分化。尽管其生物学基础复杂多样，但40多年来AML的诱导治疗一直相当统一，大多数患者的治疗效果仍然很差。最近，对白血病表观基因组的深入了解促使人们对目前处于临床开发不同阶段的多种表观遗传修饰剂进行转化研究。这些新疗法基于作用机制原理，有望改善AML患者的治疗效果。鉴于该领域最近的许多进展，我们提供了一篇最新的、以临床为导向的综述，介绍用于治疗AML的表观遗传修饰剂的不断演变的情况。
