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.
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The article was reviewed by: L. Bergmann, Department of Internal Medicine II, J. W. Goethe University Frankfurt, Frankfurt, Germany; A. E. Eşkazan, Division of Hematology, Department of Internal Medicine, Cerrahpaşa Faculty of Medicine, Istanbul University, Istanbul, Turkey; R. L. Momparler, Service of Hematology and Oncology, Centre de Recherche, CHU Sainte-Justine, University of Montréal, Montréal, QC, Canada; G. J. Ossenkoppele, Department of Hematology, VU University Medical Center, Amsterdam, The Netherlands; V. Santini, Haematology Department, AOU Careggi University of Florence, Florence, Italy. During the peer review process, the manufacturer of decitabine was also offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.
The preparation of this review was not supported by any external funding.
Conflicts of interest
K. McKeage and K. A. Lyseng-Williamson are employees of Adis/Springer, are responsible for the article content and declare no conflicts of interest.
Additional information about this Adis Review can be found at: http://www.medengine.com/Redeem/447AF0603D5CF0D9.
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McKeage, K., Lyseng-Williamson, K.A. Decitabine in newly diagnosed acute myeloid leukaemia: a profile of its use in the EU. Drugs Ther Perspect 33, 505–514 (2017). https://doi.org/10.1007/s40267-017-0445-3