Yamauchi Takahiro
Department of Hematology and Oncology, University of Fukui.
Rinsho Ketsueki. 2018;59(10):1988-1996. doi: 10.11406/rinketsu.59.1988.
The mainstay of therapeutic modalities of acute myeloid leukemia (AML) includes intensive chemotherapies and allogeneic hematopoietic cell transplantation. The gold standard of the induction treatment is a regular dose of cytarabine plus anthracycline, and several courses of consolidation therapy are administered. Allogeneic hematopoietic cell transplantation is employed in patients with intermediate-poor risks. No new drugs have been introduced to the treatment of AML for nearly 30 years. However, in 2017, the US Food and Drug Administration approved four novel drugs for treating AML: FLT3 inhibitor midostaurin, IDH2 inhibitor enasidenib, liposomal cytarabine-daunorubicin CPX-351, and revived antibody-drug conjugate gemtuzumab ozogamicin. In Japan, several new agents are also undergoing clinical trials, including Bcl-2 inhibitor venetoclax, CDK9 inhibitor alvocidib, smoothened (SMO) inhibitor glasdegib, hypomethylating agents guadecitabine and azacitidine, NEDD8 inhibitor pevonedistat, and FLT3 inhibitors quizartinib and gilteritinib. These agents will be incorporated into the conventional 7+3 regimen or combined with hypomethylating agents or low-dose cytarabine to improve the therapeutic outcomes of AML.
急性髓系白血病（AML）治疗方式的主要支柱包括强化化疗和异基因造血细胞移植。诱导治疗的金标准是常规剂量的阿糖胞苷加蒽环类药物，并进行多个疗程的巩固治疗。异基因造血细胞移植用于中低风险患者。近30年来，AML治疗未引入新的药物。然而，2017年，美国食品药品监督管理局批准了四种治疗AML的新药：FLT3抑制剂米哚妥林、IDH2抑制剂恩杂鲁胺、脂质体阿糖胞苷-柔红霉素CPX-351以及重新启用的抗体药物偶联物吉妥单抗。在日本，几种新药也正在进行临床试验，包括Bcl-2抑制剂维奈托克、CDK9抑制剂阿沃西地布、 smoothened（SMO）抑制剂格拉斯吉布、低甲基化剂胍地西他滨和阿扎胞苷、NEDD8抑制剂pevonedistat以及FLT3抑制剂quizartinib和吉列替尼。这些药物将被纳入传统的7+3方案，或与低甲基化剂或小剂量阿糖胞苷联合使用，以改善AML的治疗效果。
