1. Iron-chelating therapy with deferasirox in transfusion-dependent, higher risk myelodysplastic syndromes: a retrospective, multicentre study.
- Author
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Musto, Pellegrino, Maurillo, Luca, Simeon, Vittorio, Poloni, Antonella, Finelli, Carlo, Balleari, Enrico, Ricco, Alessandra, Rivellini, Flavia, Cortelezzi, Agostino, Tarantini, Giuseppe, Villani, Oreste, Mansueto, Giovanna, Milella, Maria R., Scapicchio, Daniele, Marziano, Gioacchino, Breccia, Massimo, Niscola, Pasquale, Sanna, Alessandro, Clissa, Cristina, and Voso, Maria T.
- Subjects
MYELODYSPLASTIC syndromes treatment ,CHELATION therapy ,DEFERASIROX ,AZACITIDINE ,BONE marrow diseases - Abstract
Iron chelation is controversial in higher risk myelodysplastic syndromes ( HR- MDS), outside the allogeneic transplant setting. We conducted a retrospective, multicentre study in 51 patients with transfusion-dependent, intermediate-to-very high risk MDS, according to the revised international prognostic scoring system, treated with the oral iron chelating agent deferasirox ( DFX). Thirty-six patients (71%) received azacitidine concomitantly. DFX was given at a median dose of 1000 mg/day (range 375-2500 mg) for a median of 11 months (range 0·4-75). Eight patients (16%) showed grade 2-3 toxicities (renal or gastrointestinal), 4 of whom (8%) required drug interruption. Median ferritin levels decreased from 1709 μg/l at baseline to 1100 μg/l after 12 months of treatment ( P = 0·02). Seventeen patients showed abnormal transaminase levels at baseline, which improved or normalized under DFX treatment in eight cases. One patient showed a remarkable haematological improvement. At a median follow up of 35·3 months, median overall survival was 37·5 months. The results of this first survey of DFX in HR- MDS are comparable, in terms of safety and efficacy, with those observed in lower-risk MDS. Though larger, prospective studies are required to demonstrate real clinical benefits, our data suggest that DFX is feasible and might be considered in a selected cohort of HR- MDS patients. [ABSTRACT FROM AUTHOR]
- Published
- 2017
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