Doucette Kimberley, Karp Judith, Lai Catherine
Georgetown University Medical Center, Lombardi Comprehensive Cancer Center, Washington, DC, USA.
Johns Hopkins University Sidney Kimmel Comprehensive Cancer Center, Baltimore, MD, USA.
Ther Adv Hematol. 2021 May 5;12:20406207211001138. doi: 10.1177/20406207211001138. eCollection 2021.
Acute myeloid leukemia (AML) is an aggressive malignancy characterized by clonal proliferation of neoplastic immature precursor cells. AML impacts older adults and has a poor prognosis. Despite recent advances in treatment, AML is complex, with both genetic and epigenetic aberrations in the malignant clone and elaborate interactions with its microenvironment. We are now able to stratify patients on the basis of specific clinical and molecular features in order to optimize individual treatment strategies. However, our understanding of the complex nature of these molecular abnormalities continues to expand the defining characteristics of high-risk mutations. In this review, we focus on genetic and microenvironmental factors in adverse risk AML that play critical roles in leukemogenesis, including those not described in an European LeukemiaNet adverse risk group, and describe therapies that are currently in the clinical arena, either approved or under development.
急性髓系白血病（AML）是一种侵袭性恶性肿瘤，其特征为肿瘤性未成熟前体细胞的克隆性增殖。AML影响老年人，预后较差。尽管近期治疗取得了进展，但AML较为复杂，恶性克隆中存在遗传和表观遗传畸变，并与其微环境存在复杂的相互作用。我们现在能够根据特定的临床和分子特征对患者进行分层，以优化个体化治疗策略。然而，我们对这些分子异常复杂性质的理解不断扩展高危突变的定义特征。在本综述中，我们聚焦于不良风险AML中的遗传和微环境因素，这些因素在白血病发生中起关键作用，包括那些未在欧洲白血病网不良风险组中描述的因素，并描述目前临床领域中已获批或正在研发的疗法。
