Decitabine in newly diagnosed acute myeloid leukaemia: a profile of its use in the EU

Abstract

Decitabine (Dacogen®), a deoxynucleoside analogue of cytidine that selectively inhibits DNA methyltransferases, provides beneficial effects in adults with newly diagnosed acute myeloid leukaemia (AML) not eligible for standard induction chemotherapy. In DACO-016, the registrational phase 3 clinical trial in this patient population, intravenous decitabine provided a clinically meaningful improvement in median overall survival (OS) relative to treatment choice (cytarabine or best supportive care) of 2 months (7.7 vs 5.0 months), as well as significant improvements in other efficacy outcomes. After a further year of follow-up, the difference in OS between treatment groups significantly favoured decitabine over treatment choice. Decitabine was generally well tolerated with a similar safety profile to that of low-dose cytarabine and low rates of early mortality. Consistent with the clinical presentation of AML, the most frequently observed adverse events were related to myelosuppression. Real-world studies support the effectiveness and tolerability profiles of decitabine in this indication.