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急性髓系白血病转化为可靶向治疗的疾病。

Acute myeloid leukemia transformed to a targetable disease.

机构信息

Hematology-Oncology Department, Faculty of Medicine, Saint Joseph University of Beirut, Beirut, Lebanon.

出版信息

Future Oncol. 2020 May;16(14):961-972. doi: 10.2217/fon-2019-0670. Epub 2020 Apr 16.

DOI:10.2217/fon-2019-0670
PMID:32297538
Abstract

Acute myeloid leukemia (AML) is a heterogeneous neoplasm characterized by the monoclonal proliferation of immature progenitors. It is the most common acute leukemia in adults and its incidence increases with age. The standard traditional treatment in fit patients was the '3 + 7' regimen and cytarabine consolidation followed or not with allogeneic stem cell transplantation. Recently, several targeted therapies such as gemtuzumab ozogamicin targeting the CD33 AML, midostaurin, gilteritinib and crenolanib inhibiting FLT3-positive AML and ivosidenib and enasidenib blocking -mutated AML have been approved. These new drugs led to the change of the landscape of the treatment of AML and transforming this disease to a targetable one. We aimed in this paper to review the implications of each new target, the mechanisms of action of these new drugs and we discuss all the studies leading to the approval of these new drugs in their indications according to each target.

摘要

急性髓细胞白血病(AML)是一种异质性肿瘤,其特征是不成熟祖细胞的单克隆增殖。它是成人中最常见的急性白血病,其发病率随着年龄的增长而增加。适合患者的标准传统治疗是“3+7”方案和阿糖胞苷巩固治疗,随后或不进行异基因干细胞移植。最近,几种靶向治疗方法已获得批准,例如针对 CD33 AML 的吉妥珠单抗奥佐米星、米哚妥林、吉特替尼和克立替尼抑制 FLT3 阳性 AML,ivosidenib 和enasidenib 阻断 - 突变 AML。这些新药改变了 AML 的治疗格局,使这种疾病成为一种可靶向的疾病。我们旨在本文中审查每个新靶标的意义,这些新药的作用机制,并根据每个靶标讨论导致这些新药在其适应证中获得批准的所有研究。

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