Gurnari Carmelo, Voso Maria Teresa, Maciejewski Jaroslaw P, Visconte Valeria
Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH 44195, USA.
Department of Biomedicine and Prevention, University of Rome Tor Vergata, 00133 Rome, Italy.
Cancers (Basel). 2020 Feb 4;12(2):357. doi: 10.3390/cancers12020357.
Acute myeloid leukemia (AML) is a heterogeneous group of clonal disorders characterized by abnormal proliferation of undifferentiated myeloid progenitors, impaired hematopoiesis, and variable response to therapy. To date, only about 30% of adult patients with AML become long-term survivors and relapse and/or disease refractoriness are the major cause of treatment failure. Thus, this is an urgent unmet clinical need and new drugs are envisaged in order to ameliorate disease survival outcomes. Here, we review the latest therapeutic approaches (investigational and approved agents) for AML treatment. A specific focus will be given to molecularly targeted therapies for AML as a representation of possible agents for precision medicine. We will discuss experimental and preclinical data for FLT3, IDH1, BCL-2, Hedgehog pathway inhibitors, and epitherapy.
急性髓系白血病（AML）是一组异质性克隆性疾病，其特征为未分化髓系祖细胞异常增殖、造血功能受损以及对治疗的反应各异。迄今为止，仅有约30%的成年AML患者成为长期幸存者，复发和/或疾病难治性是治疗失败的主要原因。因此，这是一项亟待满足的临床需求，人们设想研发新药以改善疾病生存结局。在此，我们综述AML治疗的最新治疗方法（研究性药物和已获批药物）。将特别关注AML的分子靶向治疗，作为精准医学可能使用的药物代表。我们将讨论FLT3、异柠檬酸脱氢酶1（IDH1）、B细胞淋巴瘤-2（BCL-2）、刺猬信号通路抑制剂和表观遗传治疗的实验及临床前数据。
