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Title: Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study
Authors: Porter, John ×
Galanello, Renzo
Saglio, Giuseppe
Neufeld, Ellis J
Vichinsky, Elliott
Cappellini, Maria Domenica
Olivieri, Nancy
Piga, Antonio
Cunningham, Melody J
Soulières, Denis
Gattermann, Norbert
Tchernia, Gilbert
Maertens, Johan
Giardina, Patricia
Kwiatkowski, Janet
Quarta, Giovanni
Jeng, Michael
Forni, Gian Luca
Stadler, Michael
Cario, Holger
Debusscher, Louisette
Della Porta, Matteo
Cazzola, Mario
Greenberg, Peter
Alimena, Giuliana
Rabault, Bertrand
Gathmann, Insa
Ford, John Malcolm
Alberti, Daniele
Rose, Christian #
Issue Date: Feb-2008
Publisher: Munksgaard
Series Title: European Journal of Haematology vol:80 issue:2 pages:168-176
Abstract: OBJECTIVES/METHODS: This 1-yr prospective phase II trial evaluated the efficacy of deferasirox in regularly transfused patients aged 3-81 yrs with myelodysplastic syndromes (MDS; n = 47), Diamond-Blackfan anaemia (DBA; n = 30), other rare anaemias (n = 22) or beta-thalassaemia (n = 85). Dosage was determined by baseline liver iron concentration (LIC). RESULTS: In patients with baseline LIC > or = 7 mg Fe/g dry weight, deferasirox initiated at 20 or 30 mg/kg/d produced statistically significant decreases in LIC (P < 0.001); these decreases were greatest in MDS and least in DBA. As chelation efficiency and iron excretion did not differ significantly between disease groups, the differences in LIC changes are consistent with mean transfusional iron intake (least in MDS: 0.28 +/- 0.14 mg/kg/d; greatest in DBA: 0.4 +/- 0.11 mg/kg/d). Overall, LIC changes were dependent on dose (P < 0.001) and transfusional iron intake (P < 0.01), but not statistically different between disease groups. Changes in serum ferritin and LIC were correlated irrespective of disease group (r = 0.59), supporting the potential use of serum ferritin for monitoring deferasirox therapy. Deferasirox had a safety profile compatible with long-term use. There were no disease-specific safety/tolerability effects: the most common adverse events were gastrointestinal disturbances, skin rash and non-progressive serum creatinine increases. CONCLUSIONS: Deferasirox is effective for reducing iron burden with a defined, clinically manageable safety profile in patients with various transfusion-dependent anaemias. There were no disease-specific adverse events. Once differences in transfusional iron intake are accounted for, dose-dependent changes in LIC or serum ferritin are similar in MDS and other disease groups.
URI: 
ISSN: 0902-4441
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Hematology Section (-)
× corresponding author
# (joint) last author

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