Azacitidine for the treatment of lower risk myelodysplastic syndromes: A retrospective study of 74 patients enrolled in an Italian named patient program

Pellegrino Musto, Luca Maurillo, Alessandra Spagnoli, Antonella Gozzini, Flavia Rivellini, Monia Lunghi, Oreste Villani, Maria Antonietta Aloe-Spiriti, Adriano Venditti, Valeria Santini, Giuseppe Leone, Maria Teresa Voso, Alfonso Maria D'Arco, Caterina Tatarelli, Dario Ferrero, Gianluca Gaidano, Giuseppe Palumbo, Francesco Di Raimondo, Esther Oliva, Grazia SanpaoloAnna Tonso, Alberto Santagostino, Nunzio Filardi, Berardino Pollio, Anna Candoni, Carla Fili, Domenico Russo, Enrico Orciuolo, Mario Petrini, Lucia Ciuffreda, Antonio Riezzo, Fortunato Morabito, Patrizio Mazza, Domenico Pastore, Giorgina Specchia, Felicetto Ferrara

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)1485-1494
Number of pages10
Issue number6
Publication statusPublished - Mar 15 2010


  • Azacitidine
  • Hypomethylating agents
  • International prognostic
  • Myelodysplastic syndromes
  • Prognosis
  • Scoring system
  • Transfusion

ASJC Scopus subject areas

  • Cancer Research
  • Oncology


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