Azacitidine for the treatment of lower risk myelodysplastic syndromes : a retrospective study of 74 patients enrolled in an Italian named patient program
| Autor: | Musto, Pellegrino, Maurillo, Luca, Spagnoli, Alessandra, Gozzini, Antonella, Rivellini, Flavia, Lunghi, Monia, Villani, Oreste, Aloe Spiriti, Maria Antonietta, Venditti, Adriano, Santini, Valeria, Leone, Giuseppe, Voso, Maria Teresa, D'Arco, Alfonso Maria, Tatarelli, Caterina, Ferrero, Dario, Gaidano, Gianluca, Palumbo, Giuseppe, Di Raimondo, Francesco, Oliva, Esther, Sanpaolo, Grazia, Tonso, Anna, Santagostino, Alberto, Filardi, Nunzio, Pollio, Berardino, Candoni, Anna, Fili, Carla, Russo, Domenico, Orciuolo, Enrico, Petrini, Mario, Ciuffreda, Lucia, Riezzo, Antonio, Morabito, Fortunato, Mazza, Patrizio, Pastore, Domenico, Specchia, Giorgina, Ferrara, Felicetto |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2010 |
| Předmět: |
Azacitidine
Hypomethylating agents International prognostic Myelodysplastic syndromes Prognosis Scoring system Transfusion Adult Aged Aged 80 and over Antimetabolites Antineoplastic Drug Evaluation Female Humans Italy Male Middle Aged Myelodysplastic Syndromes Retrospective Studies Risk Treatment Outcome Cancer Research Oncology Antimetabolites 80 and over hypomethylating agents Antineoplastic myelodysplastic syndromes International Prognostic Scoring System medicine.drug medicine.medical_specialty azacitidine medicine.drug_class Lower risk Antimetabolite Internal medicine azacitidine hypomethylating agents myelodysplastic syndromes prognosis International Prognostic Scoring System transfusion medicine Risk factor Adverse effect transfusion business.industry Retrospective cohort study medicine.disease Surgery prognosis business Settore MED/15 - Malattie del Sangue |
| Popis: | 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. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|