title: Deferasirox chelation therapy in patients with transfusion-dependent MDS: a 'real-world' report from two regional Italian registries: Gruppo Romano Mielodisplasie and Registro Basilicata.
abstract: Deferasirox (DFX) is an orally administered iron chelator approved for use in patients with transfusion-dependent iron overload due to myelodysplastic syndromes (MDS). The safety and efficacy of DFX has been explored in clinical trial settings, but there is little data on unselected patients with MDS. The aim of this study was to retrospectively evaluate the safety, compliance, efficacy and effect on haematopoiesis of DFX in a large 'real-world' MDS population. One hundred and eighteen patients with transfusion-dependent MDS were treated with DFX across 11 centres in Italy. Serum ferritin levels, haematological response, dosing, adverse events and transfusion dependence were recorded at baseline, 3, 6, 12 and 24 months following initiation of treatment. DFX reduced mean serum ferritin levels from 1790 to 1140 ng/mL (P < 0.001), with 7.1% of patients achieving transfusion independence. Significant haematological improvement was seen in erythroid (17.6%), platelet (5.9%) and neutrophil counts (7.1%). Adverse events were reported in 47.5% of patients, including gastrointestinal and renal toxicity. Regression analysis showed that higher starting doses of DFX are associated with transfusion independence at 24 months. DFX is a safe, effective treatment for transfusion-dependent MDS that can lead to transfusion independence and haematological improvement in a subset of patients.
authors.affiliation: Ematologia, Fondazione Policlinico Tor Vergata, Rome, Italy.
authors.forename: Luca
authors.initials: L
authors.lastname: Maurillo
authors.affiliation: Ematologia, Università la "Sapienza", Rome, Italy.
authors.forename: Massimo
authors.initials: M
authors.lastname: Breccia
authors.affiliation: Ematologia, Fondazione Policlinico Tor Vergata, Rome, Italy.|Ematologia, Dipartimento di Biomedicina e Prevenzione, Università Tor Vergata, Rome, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Buccisano
authors.affiliation: Ematologia, Fondazione Policlinico Tor Vergata, Rome, Italy.|Ematologia, Dipartimento di Biomedicina e Prevenzione, Università Tor Vergata, Rome, Italy.
authors.forename: Maria Teresa
authors.initials: MT
authors.lastname: Voso
authors.affiliation: UOC Ematologia, Ospedale S.Eugenio, Rome, Italy.
authors.forename: Pasquale
authors.initials: P
authors.lastname: Niscola
authors.affiliation: UOC Ematologia, Ospedale di Belcolle, Viterbo, Italy.
authors.forename: Giulio
authors.initials: G
authors.lastname: Trapè
authors.affiliation: Ematologia, AOU S. Andrea, Rome, Italy.
authors.forename: Caterina
authors.initials: C
authors.lastname: Tatarelli
authors.affiliation: UOC Immunoematologia e Medicina Trasfusionale, Osp. S.Pietro Fatebenefratelli, Rome, Italy.
authors.forename: Ada
authors.initials: A
authors.lastname: D'Addosio
authors.affiliation: Ematologia, Università la "Sapienza", Rome, Italy.
authors.forename: Roberto
authors.initials: R
authors.lastname: Latagliata
authors.affiliation: UOC Ematologia, AO S.Giovanni, Rome, Italy.
authors.forename: Susanna
authors.initials: S
authors.lastname: Fenu
authors.affiliation: Ematologia, Osp. S. Pertini, Rome, Italy.
authors.forename: Anna Lina
authors.initials: AL
authors.lastname: Piccioni
authors.affiliation: UOS Ematologia, Presidio Ospedieliero Madonna delle Grazie, Matera, Italy.
authors.forename: Alberto
authors.initials: A
authors.lastname: Fragasso
authors.affiliation: Ematologia, AOU S. Andrea, Rome, Italy.
authors.forename: Maria A
authors.initials: MA
authors.lastname: Aloe Spiriti
authors.affiliation: Ematologia, Fondazione Policlinico Tor Vergata, Rome, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Refrigeri
authors.affiliation: Ematologia, Università Cattolica Sacro Cuore, Rome, Italy.
authors.forename: Marianna
authors.initials: M
authors.lastname: Criscuolo
authors.affiliation: Ematologia, Università Cattolica Sacro Cuore, Rome, Italy.|Oncoematologia, IRCCS, Centro di Riferimento Oncologico della Basilicata, Rionero in Vulture, Italy.
authors.forename: Pellegrino
authors.initials: P
authors.lastname: Musto
authors.affiliation: Ematologia, Fondazione Policlinico Tor Vergata, Rome, Italy.|Ematologia, Dipartimento di Biomedicina e Prevenzione, Università Tor Vergata, Rome, Italy.
authors.forename: Adriano
authors.initials: A
authors.lastname: Venditti
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001565:Benzoates / Q000627:therapeutic use*; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000097:blood; D006410:Hematopoiesis / Q000187:drug effects; D006801:Humans; D007501:Iron / Q000097:blood; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D007558:Italy; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000097:blood / Q000473:pathology / Q000628:therapy*; D012042:Registries; D012189:Retrospective Studies; D065227:Transfusion Reaction; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000077588:Deferasirox
keywords: deferasirox; efficacy; erythroid response; iron chelation; myelodysplastic syndromes; safety