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 ¼ 29 patients), 7 days (n ¼ 43 patients), or 10 days (n ¼ 2 patients) every month at a dose of 75 mg/m2 daily (n ¼ 45 patients) or at a fixed dose of 100 mg daily (n ¼ 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 at least 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.
PELLEGRINO, M., MAURILLO, L., SPAGNOLI, A., GOZZINI, A., RIVELLINI, F., LUNGHI, M., et al. (2010). Azacitidine for the treatment of lower risk myelodysplastic syndromes : A retrospective study of 74 patients enrolled in an Italian named patient program. CANCER, 116, 1485-1494.
|Tipologia:||Articolo su rivista|
|Citazione:||PELLEGRINO, M., MAURILLO, L., SPAGNOLI, A., GOZZINI, A., RIVELLINI, F., LUNGHI, M., et al. (2010). Azacitidine for the treatment of lower risk myelodysplastic syndromes : A retrospective study of 74 patients enrolled in an Italian named patient program. CANCER, 116, 1485-1494.|
|IF:||Con Impact Factor ISI|
|Settore Scientifico Disciplinare:||Settore MED/15 - Malattie del Sangue|
|Revisione (peer review):||Sì, ma tipo non specificato|
|Digital Object Identifier (DOI):||http://dx.doi.org/10.1002/cncr.24894|
|Stato di pubblicazione:||Pubblicato|
|Data di pubblicazione:||2010|
|Titolo:||Azacitidine for the treatment of lower risk myelodysplastic syndromes : A retrospective study of 74 patients enrolled in an Italian named patient program.|
|Autori:||PELLEGRINO,M; MAURILLO,L; SPAGNOLI,A; GOZZINI,A; RIVELLINI,F; LUNGHI,M; VILLANI,O; ALOE-SPIRITI,MA; VENDITTI, A; SANTINI,V.|
|Appare nelle tipologie:||01 - Articolo su rivista|