TY - JOUR
T1 - Deferasirox chelation therapy in patients with transfusion-dependent MDS
T2 - A 'real-world' report from two regional Italian registries: Gruppo Romano Mielodisplasie and Registro Basilicata
AU - Maurillo, Luca
AU - Breccia, Massimo
AU - Buccisano, Francesco
AU - Voso, Maria Teresa
AU - Niscola, Pasquale
AU - Trapè, Giulio
AU - Tatarelli, Caterina
AU - D'Addosio, Ada
AU - Latagliata, Roberto
AU - Fenu, Susanna
AU - Piccioni, Anna Lina
AU - Fragasso, Alberto
AU - Aloe Spiriti, Maria A.
AU - Refrigeri, Marco
AU - Criscuolo, Marianna
AU - Musto, Pellegrino
AU - Venditti, Adriano
PY - 2015/7/1
Y1 - 2015/7/1
N2 - 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.
AB - 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.
KW - Deferasirox
KW - Efficacy
KW - Erythroid response
KW - Iron chelation
KW - Myelodysplastic syndromes
KW - Safety
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U2 - 10.1111/ejh.12476
DO - 10.1111/ejh.12476
M3 - Article
C2 - 25764148
AN - SCOPUS:84931574624
VL - 95
SP - 52
EP - 56
JO - European Journal of Haematology
JF - European Journal of Haematology
SN - 0902-4441
IS - 1
ER -