Azacitidine for the treatment of lower risk myelodysplastic syndromes

A retrospective study of 74 patients enrolled in an Italian named patient program


  • Presented in part as an oral presentation at the 13th Congress of the European Hematology Association, Copenhagen, Denmark, June 12-15, 2008; and as a poster presentation at the 50th Annual Meeting of the American Society of Hematology, San Francisco, California, December 6-9, 2008.

  • The following are members of the Italian Cooperative Study Group on Azacitidine in Myelodysplastic Syndromes and Acute Leukemias: Giuseppe Leone and Maria Teresa Voso, Chair of Hematology, “Sacro Cuore” Catholic University, Rome; Alfonso Maria D'Arco, Department of Onco-Hematology, “Umberto I” Hospital, Nocera, Salerno; Caterina Tatarelli, “S. Andrea” Hospital, Rome; Dario Ferrero, Chair of Hematology, University of Turin, Turin; Gianluca Gaidano, Department of Hematology, “A. Avogadro” University, Novara; Giuseppe Palumbo and Francesco Di Raimondo, Chair of Hematology, University of Catania, Catania; Esther Oliva, Department of Hematology, “Bianchi-Melacrino-Morelli” Hospital, Reggio Calabria; Grazia Sanpaolo, Department of Hematology, Scientific Institute of Research and Cure, “Casa Sollievo della Sofferenza” Hospital, S. Giovanni Rotondo, Foggia; Anna Tonso, Internal Medicine, Civic Hospital, Biella; Alberto Santagostino, Department of Internal Medicine, Civic Hospital, Vercelli; Nunzio Filardi, Department of Hematology, “S. Carlo” Hospital, Potenza; Berardino Pollio, Department of Hematology and Transfusion Service, Civic Hospital, Ivrea; Anna Candoni, Chair of Hematology, University of Udine, Udine; Carla Fili and Domenico Russo, Chair of Hematology, University of Brescia, Brescia; Enrico Orciuolo and Mario Petrini, Chair of Hematology, University of Pisa, Pisa; Lucia Ciuffreda and Antonio Riezzo, Department of Hematology, “S. Nicola Pellegrino” Hospital, Trani; Fortunato Morabito, Department of Hematology, “Annunziata” Hospital, Cosenza; Patrizio Mazza, Department of Hematology, “Moscati” Hospital, Taranto; Domenico Pastore and Giorgina Specchia, Chair of Hematology, University of Bari, Bari; and Felicetto Ferrara, Department of Hematology, “A. Cardarelli” Hospital, Naples, Italy.



Azacitidine induces responses and prolongs overall survival compared with conventional care regimens in patients who have high-risk myelodysplastic syndromes (MDS). However, limited data are available concerning the efficacy and safety of azacitidine in patients who have lower risk MDS.


The authors retrospectively evaluated 74 patients with International Prognostic Scoring System low-risk or intermediate 1-risk MDS, who received azacitidine on a national named patient program. At baseline, 84% of patients were transfusion-dependent, 57% had received erythropoietin, and 51% were aged >70 years. Azacitidine was administered subcutaneously for 5 days (n = 29 patients), 7 days (n = 43 patients), or 10 days (n = 2 patients) every month at a dose of 75 mg/m2 daily (n = 45 patients) or at a fixed dose of 100 mg daily (n = 29 patients) and for a median of 7 cycles (range, 1-30 cycles).


According to the 2006 International Working Group criteria, overall response rate (ORR) was 45.9%, including complete responses (10.8%), partial responses (9.5%), hematologic improvements (20.3%), and bone marrow complete responses (5.4%). The ORR was 51.6% in 64 patients who completed ≥4 cycles of treatment. The median duration of response was 6 months (range, 1-30 months). After a median follow-up of 15 months, 71% of patients remained alive. A survival benefit was observed in responders versus nonresponders (94% vs 54% of patients projected to be alive at 2.5 years, respectively; P < .0014). The most common grade 3 or 4 adverse events were myelosuppression (21.6%) and infection (6.8%).


The current results indicated that azacitidine may be a feasible and effective treatment for patients with lower risk MDS. Cancer 2010. © 2010 American Cancer Society.