Azacitidine for the treatment of lower risk myelodysplastic syndromes

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

Authors


  • 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.

Abstract

BACKGROUND:

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.

METHODS:

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).

RESULTS:

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%).

CONCLUSIONS:

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.

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