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 mu g/l at baseline to 1100 mu g/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.

Musto, P., Maurillo, L., Simeon, V., Poloni, A., Finelli, C., Balleari, E., et al. (2017). Iron-chelating therapy with deferasirox in transfusion-dependent, higher risk myelodysplastic syndromes: a retrospective, multicentre study. BRITISH JOURNAL OF HAEMATOLOGY, 177(5), 741-750 [10.1111/bjh.14621].

Iron-chelating therapy with deferasirox in transfusion-dependent, higher risk myelodysplastic syndromes: a retrospective, multicentre study

Voso M. T.;Venditti A.;
2017-06

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 mu g/l at baseline to 1100 mu g/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.
Pubblicato
Rilevanza internazionale
Articolo
Esperti anonimi
Settore MED/15 - Malattie del Sangue
English
Con Impact Factor ISI
International Prognostic Scoring System; deferasirox; iron chelation; myelodyslastic syndromes; revised-International Prognostic Scoring System; Administration, Oral; Adult; Aged; Aged, 80 and over; Benzoates; Chelation Therapy; Erythrocyte Transfusion; Female; Ferritins; Humans; Iron Chelating Agents; Male; Middle Aged; Myelodysplastic Syndromes; Retrospective Studies; Treatment Outcome; Triazoles
Musto, P., Maurillo, L., Simeon, V., Poloni, A., Finelli, C., Balleari, E., et al. (2017). Iron-chelating therapy with deferasirox in transfusion-dependent, higher risk myelodysplastic syndromes: a retrospective, multicentre study. BRITISH JOURNAL OF HAEMATOLOGY, 177(5), 741-750 [10.1111/bjh.14621].
Musto, P; Maurillo, L; Simeon, V; Poloni, A; Finelli, C; Balleari, E; Ricco, A; Rivellini, F; Cortelezzi, A; Tarantini, G; Villani, O; Mansueto, G; Milella, Mr; Scapicchio, D; Marziano, G; Breccia, M; Niscola, P; Sanna, A; Clissa, C; Voso, Mt; Fenu, S; Venditti, A; Santini, V; Angelucci, E; Levis, A
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Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/2108/194341
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