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. © 2010 American Cancer Society.

Azacitidine for the treatment of lower risk myelodysplastic syndromes: A retrospective study of 74 patients enrolled in an Italian named patient program / Musto, P.; Maurillo, L.; Spagnoli, A.; Gozzini, A.; Rivellini, F.; Lunghi, M.; Villani, O.; Aloe-Spiriti, M. A.; Venditti, A.; Santini, V.; Leone, G.; Voso, M. T.; D'Arco, A. M.; Tatarelli, C.; Ferrero, D.; Gaidano, G.; Palumbo, G.; Di Raimondo, F.; Oliva, E.; Sanpaolo, G.; Tonso, A.; Santagostino, A.; Filardi, N.; Pollio, B.; Candoni, A.; Fili, C.; Russo, D.; Orciuolo, E.; Petrini, M.; Ciuffreda, L.; Riezzo, A.; Morabito, F.; Mazza, P.; Pastore, D.; Specchia, G.; Ferrara, F.. - In: CANCER. - ISSN 0008-543X. - 116:6(2010), pp. 1485-1494. [10.1002/cncr.24894]

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

Candoni A.;
2010-01-01

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. © 2010 American Cancer Society.
116
6
1485
1494
Azacitidine for the treatment of lower risk myelodysplastic syndromes: A retrospective study of 74 patients enrolled in an Italian named patient program / Musto, P.; Maurillo, L.; Spagnoli, A.; Gozzini, A.; Rivellini, F.; Lunghi, M.; Villani, O.; Aloe-Spiriti, M. A.; Venditti, A.; Santini, V.; Leone, G.; Voso, M. T.; D'Arco, A. M.; Tatarelli, C.; Ferrero, D.; Gaidano, G.; Palumbo, G.; Di Raimondo, F.; Oliva, E.; Sanpaolo, G.; Tonso, A.; Santagostino, A.; Filardi, N.; Pollio, B.; Candoni, A.; Fili, C.; Russo, D.; Orciuolo, E.; Petrini, M.; Ciuffreda, L.; Riezzo, A.; Morabito, F.; Mazza, P.; Pastore, D.; Specchia, G.; Ferrara, F.. - In: CANCER. - ISSN 0008-543X. - 116:6(2010), pp. 1485-1494. [10.1002/cncr.24894]
Musto, P.; Maurillo, L.; Spagnoli, A.; Gozzini, A.; Rivellini, F.; Lunghi, M.; Villani, O.; Aloe-Spiriti, M. A.; Venditti, A.; Santini, V.; Leone, G.; Voso, M. T.; D'Arco, A. M.; Tatarelli, C.; Ferrero, D.; Gaidano, G.; Palumbo, G.; Di Raimondo, F.; Oliva, E.; Sanpaolo, G.; Tonso, A.; Santagostino, A.; Filardi, N.; Pollio, B.; Candoni, A.; Fili, C.; Russo, D.; Orciuolo, E.; Petrini, M.; Ciuffreda, L.; Riezzo, A.; Morabito, F.; Mazza, P.; Pastore, D.; Specchia, G.; Ferrara, F.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11380/1294110
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