Research has resulted in regulatory approval of nine agents for acute myeloid leukaemia indications by the US Food and Drug Administration since 2017: the Bcl-2 inhibitor, venetoclax; two FLT3 inhibitors, midostaurin and gilteritinib; two IDH inhibitors, ivosidenib (IDH1 inhibitor) and enasidenib (IDH2 inhibitor); the anti-CD33 antibody-drug conjugate, gemtuzumab ozogamicin; the oral, poorly absorbable hypomethylating agent, azacitidine; the liposomal formulation of cytarabine and daunorubicin (5:1 ratio), CPX-351; and the hedgehog signalling pathway inhibitor, glasdegib. A 100% absorbable oral formulation of the hypomethylating agent decitabine was approved for the treatment of myelodysplastic syndrome and chronic myelomonocytic leukaemia, and might be used as an alternative to parenteral hypomethylating agents. Several of the approvals are as single-agent therapies or in specific combinations for narrow indications, thus offering poor treatment value. In this Review, we discuss ongoing research into combinations containing these commercially available targeted therapies for acute myeloid leukaemia.

中文翻译：

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利用现有靶向疗法治疗急性髓系白血病的益处。


自 2017 年以来，研究已导致美国食品和药物管理局批准了九种用于治疗急性髓性白血病适应症的药物：Bcl-2 抑制剂 Venetoclax；两种FLT3抑制剂，米斯托林和gilteritinib；两种IDH抑制剂，ivosidenib（IDH1抑制剂）和enasidenib（IDH2抑制剂）；抗CD33抗体-药物缀合物，吉妥珠单抗奥佐米星；口服、吸收性差的低甲基化剂阿扎胞苷；阿糖胞苷和柔红霉素的脂质体制剂(5:1比例)，CPX-351；以及刺猬信号通路抑制剂 glasdegib。 100%可吸收的口服低甲基化剂地西他滨被批准用于治疗骨髓增生异常综合征和慢性粒单核细胞白血病，并可用作肠外低甲基化剂的替代品。其中一些批准的药物是作为单药疗法或针对狭窄适应症的特定组合疗法，因此治疗价值较差。在这篇综述中，我们讨论了正在进行的针对包含这些市售靶向治疗急性髓系白血病的组合的研究。