Publication: Tailoring iron chelation by iron intake and serum ferritin: The prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias
Issued Date
2010-04-01
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15928721
03906078
03906078
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2-s2.0-77950682176
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Mahidol University
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SCOPUS
Bibliographic Citation
Haematologica. Vol.95, No.4 (2010), 557-566
Suggested Citation
Maria Domenica Cappellini, John Porter, Amal El-Beshlawy, Chi Kong Li, John F. Seymour, Mohsen Elalfy, Norbert Gattermann, Stéphane Giraudier, Jong Wook Lee, Lee Lee Chan, Kai Hsin Lin, Christian Rose, Ali Taher, Swee Lay Thein, Vip Viprakasit, Dany Habr, Gabor Domokos, Bernard Roubert, Antonis Kattamis Tailoring iron chelation by iron intake and serum ferritin: The prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias. Haematologica. Vol.95, No.4 (2010), 557-566. doi:10.3324/haematol.2009.014696 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/29730
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Title
Tailoring iron chelation by iron intake and serum ferritin: The prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias
Other Contributor(s)
Universita degli Studi di Milano
UCL
Cairo University
Prince of Wales Hospital Hong Kong
Peter Maccallum Cancer Centre
Ain Shams University
Heinrich Heine Universitat
Hopital Henri Mondor
The Catholic University of Korea
University of Malaya Medical Centre
National Taiwan University Hospital
Hopital Saint-Vincent-de-Paul
American University of Beirut
King's College London
Mahidol University
Novartis Pharmaceuticals Corporation
Novartis International AG
University of Athens
UCL
Cairo University
Prince of Wales Hospital Hong Kong
Peter Maccallum Cancer Centre
Ain Shams University
Heinrich Heine Universitat
Hopital Henri Mondor
The Catholic University of Korea
University of Malaya Medical Centre
National Taiwan University Hospital
Hopital Saint-Vincent-de-Paul
American University of Beirut
King's College London
Mahidol University
Novartis Pharmaceuticals Corporation
Novartis International AG
University of Athens
Abstract
Background: Following a clinical evaluation of deferasirox (Exjade®) it was concluded that, in addition to baseline body iron burden, ongoing transfusional iron intake should be considered when selecting doses. The 1-year EPIC study, the largest ever investigation conducted for an iron chelator, is the first to evaluate whether fixed starting doses of deferasirox, based on transfusional iron intake, with dose titration guided by serum ferritin trends and safety markers, provides clinically acceptable chelation in patients (aged ≥2 years) with transfusional hemosiderosis from various types of anemia. Design and Methods: The recommended initial dose was 20 mg/kg/day for patients receiving 2-4 packed red blood cell units/month and 10 or 30 mg/kg/day was recommended for patients receiving less or more frequent transfusions, respectively. Dose adjustments were based on 3-month serum ferritin trends and continuous assessment of safety markers. The primary efficacy end-point was change in serum ferritin after 52 weeks compared with baseline. Results: The 1744 patients enrolled had the following conditions; thalassemia (n=1115), myelodysplastic syndromes (n=341), aplastic anemia (n=116), sickle cell disease (n=80), rare anemias (n=43) and other transfused anemias (n=49). Overall, there was a significant reduction in serum ferritin from baseline (-264 ng/mL; P<0.0001), reflecting dosage adjustments and ongoing iron intake. The most common (>5%) adverse events were gastrointestinal disturbances (28%) and skin rash (10%). Conclusions: Analysis of this large, prospectively collected data set confirms the response to chelation ther- apy across various anemias, supporting initial deferasirox doses based on transfusional iron intake, with subsequent dose titration guided by trends in serum ferritin and safety markers (clinicaltrials.gov identifier: NCT00171821). © 2010 Ferrata Storti Foundation.