Efficacy of 5-azacytidine, a hypomethylating agent, in patients with myeloproliferative neoplasm in accelerated or blast crisis phase


  • S.A. Guseva Shupyk National Medical Academy of Postgraduate Education, Kyiv, Ukraine,
  • Ya.P. Goncharov National Military Clinical Medical Center “Main Military Clinical Hospital”, Kyiv, Ukraine,
  • E.V. Kucher Shupyk National Medical Academy of Postgraduate Education, Kyiv, Ukraine,




сhronic myeloproliferative neoplasms, myelofibrosis, 5-azacytidine, hypomethylating agents, efficacy


Chronic myeloproliferative neoplasms (MPNs) transformed into acute myeloid leukemia/myelodysplastic syndrome, MPNs in accelerated phase and high-risk primary myelofibrosis are associated with poor response to chemotherapy and very short survival. Epigenetic changes play an important role in cancer pathogenesis through transcriptional silencing of critical tumor suppressor genes. Data on the activity of DNA methyltransferase inhibitor, 5-azacytidine, in patients with MPN were published. Fifty-four patients with Ph-negative MPN (including 21 persons with essential thrombocythemia, 21 — with polycythemia vera, 7 — with primary myelofibrosis, and 5 — with unclassified MPN) had progressed to acute myeloid leukemia (n = 26) or myelodysplastic syndrome (n = 28). They were treated with 5-azacytidine at a daily dose of 75 mg/m2 subcutaneously during 7 days every 28 days according to the schedule approved by the Food and Drug Administration/European Medicines Evaluation Agency. Overall response rate was 52 % (24 % — complete response, 11 % — partial response, 8 % — bone marrow, or incomplete recovery with cytopenia, 9 % — hematolo­gic improvement), and median response duration was 9 months. Recurrence of chronic phase of the initial MPN was observed in 39 % of the responders. Median overall survival was 11 months. Myelosuppression was the major adverse effect, with grade 3–4 neutropenia in 10 (29 %) patients. The first own positive experience of using 5-azacytidine (Vinduza, Reddy’s Laboratories) in patient C. with the blast crisis is presented. Partial remission developed in this patient after 3 courses of 5-azacytidine and persisted for 4 months.


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