Maintenance treatment with azacytidine for patients with high-risk myelodysplastic syndromes (MDS) or acute myeloid leukaemia following MDS in complete remission after induction chemotherapy

Research output: Contribution to journalArticle


P>This prospective Phase II study is the first to assess the feasibility and efficacy of maintenance 5-azacytidine for older patients with high-risk myelodysplastic syndrome (MDS), chronic myelomonocytic leukaemia and MDS-acute myeloid leukaemia syndromes in complete remission (CR) after induction chemotherapy. Sixty patients were enrolled and treated by standard induction chemotherapy. Patients that reached CR started maintenance therapy with subcutaneous azacytidine, 5/28 d until relapse. Promoter-methylation status of CDKN2B (P15 ink4b), CDH1 and HIC1 was examined pre-induction, in CR and 6, 12 and 24 months post CR. Twenty-four (40%) patients achieved CR after induction chemotherapy and 23 started maintenance treatment with azacytidine. Median CR duration was 13 center dot 5 months, > 24 months in 17% of the patients, and 18-30 center dot 5 months in the four patients with trisomy 8. CR duration was not associated with CDKN2B methylation status or karyotype. Median overall survival was 20 months. Hypermethylation of CDH1 was significantly associated with low CR rate, early relapse, and short overall survival (P = 0 center dot 003). 5-azacytidine treatment, at a dose of 60 mg/m2 was well tolerated. Grade III-IV thrombocytopenia and neutropenia occurred after 9 center dot 5 and 30% of the cycles, respectively, while haemoglobin levels increased during treatment. 5-azacytidine treatment is safe, feasible and may be of benefit in a subset of patients.


  • Michael Grovdal
  • Mohsen Karimi
  • Rasheed Khan
  • Anni Aggerholm
  • Petar Antunovic
  • Jan Astermark
  • Per Bernell
  • Lena-Maria Engstrom
  • Lars Kjeldsen
  • Olle Linder
  • Lars Nilsson
  • Anna Olsson
  • Mette S. Holm
  • Jon M. Tangen
  • Jonas Wallvik
  • Gunnar Oberg
  • Peter Hokland
  • Sten Eirik W Jacobsen
  • Anna Porwit
  • Eva Hellstrom-Lindberg
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Hematology


  • therapy, maintenance, clinical studies, myelodysplastic syndrome, azacytidine, DNA-methylation
Original languageEnglish
Pages (from-to)293-302
JournalBritish Journal of Haematology
Issue number3
Publication statusPublished - 2010
Publication categoryResearch