TY - JOUR
T1 - Azacitidine for the treatment of lower risk myelodysplastic syndromes
T2 - A retrospective study of 74 patients enrolled in an Italian named patient program
AU - Musto, Pellegrino
AU - Maurillo, Luca
AU - Spagnoli, Alessandra
AU - Gozzini, Antonella
AU - Rivellini, Flavia
AU - Lunghi, Monia
AU - Villani, Oreste
AU - Aloe-Spiriti, Maria Antonietta
AU - Venditti, Adriano
AU - Santini, Valeria
AU - Leone, Giuseppe
AU - Voso, Maria Teresa
AU - D'Arco, Alfonso Maria
AU - Tatarelli, Caterina
AU - Ferrero, Dario
AU - Gaidano, Gianluca
AU - Palumbo, Giuseppe
AU - Di Raimondo, Francesco
AU - Oliva, Esther
AU - Sanpaolo, Grazia
AU - Tonso, Anna
AU - Santagostino, Alberto
AU - Filardi, Nunzio
AU - Pollio, Berardino
AU - Candoni, Anna
AU - Fili, Carla
AU - Russo, Domenico
AU - Orciuolo, Enrico
AU - Petrini, Mario
AU - Ciuffreda, Lucia
AU - Riezzo, Antonio
AU - Morabito, Fortunato
AU - Mazza, Patrizio
AU - Pastore, Domenico
AU - Specchia, Giorgina
AU - Ferrara, Felicetto
PY - 2010/3/15
Y1 - 2010/3/15
N2 - 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.
AB - 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.
KW - Azacitidine
KW - Hypomethylating agents
KW - International prognostic
KW - Myelodysplastic syndromes
KW - Prognosis
KW - Scoring system
KW - Transfusion
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U2 - 10.1002/cncr.24894
DO - 10.1002/cncr.24894
M3 - Article
C2 - 20151429
AN - SCOPUS:77949364616
VL - 116
SP - 1485
EP - 1494
JO - Cancer
JF - Cancer
SN - 0008-543X
IS - 6
ER -