Georgetown University Medical Center, Lombardi Comprehensive Cancer Center, Washington, DC, USA.
Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, USA.
J Hematol Oncol. 2019 Sep 18;12(1):100. doi: 10.1186/s13045-019-0774-x.
Acute myeloid leukemia (AML) is the most common form of acute leukemia in adults, with an incidence that increases with age, and a generally poor prognosis. The disease is clinically and genetically heterogeneous, and recent advances have improved our understanding of the cytogenetic abnormalities and molecular mutations, aiding in prognostication and risk stratification. Until recently, however, therapeutic options were mostly limited to cytotoxic chemotherapy. Since 2017, there has been an explosion of newly approved treatment options both nationally and internationally, with the majority of new drugs targeting specific gene mutations and/or pivotal cell survival pathways. In this review article, we will discuss these new agents approved for the treatment of AML within the last 2 years, and will outline the mechanistic features and clinical trials that led to their approvals.
急性髓系白血病(AML)是成人中最常见的急性白血病形式,发病率随年龄增长而增加,总体预后较差。该疾病在临床上和遗传学上具有异质性,最近的进展提高了我们对细胞遗传学异常和分子突变的认识,有助于预后判断和风险分层。然而,直到最近,治疗选择主要限于细胞毒性化疗。自 2017 年以来,国内外新批准的治疗方案大量涌现,大多数新药针对特定的基因突变和/或关键的细胞存活途径。在这篇综述文章中,我们将讨论过去 2 年内批准用于治疗 AML 的这些新药物,并概述导致其批准的机制特征和临床试验。
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