For the past 40 years, the standard of care for acute myeloid leukemia (AML) has been cytotoxic chemotherapy of cytarabine and an anthracycline. The year 2017 has seen milestones in AML treatment with the US Food and Drug Administration (FDA) approval of novel therapies, including the anti–CD33-calicheamicin conjugate gemtuzumab ozogamicin, a liposome-encapsulated formulation of daunorubicin-cytarabine, the FLT3 inhibitor midostaurin, and the mutant isocitrate dehydrogenase 2 (mIDH2) inhibitor enasidenib mesylate. The FDA approval of enasidenib in August 2017 represents a critical breakthrough for targeted therapy and precision medicine for AML.
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