TY - JOUR
T1 - Tailoring iron chelation by iron intake and serum ferritin
T2 - The prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias
AU - Cappellini, Maria Domenica
AU - Porter, John
AU - El-Beshlawy, Amal
AU - Li, Chi Kong
AU - Seymour, John F.
AU - Elalfy, Mohsen
AU - Gattermann, Norbert
AU - Giraudier, Stéphane
AU - Lee, Jong Wook
AU - Chan, Lee Lee
AU - Lin, Kai Hsin
AU - Rose, Christian
AU - Taher, Ali
AU - Thein, Swee Lay
AU - Viprakasit, Vip
AU - Habr, Dany
AU - Domokos, Gabor
AU - Roubert, Bernard
AU - Kattamis, Antonis
PY - 2010/4
Y1 - 2010/4
N2 - 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; P5%) 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).
AB - 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; P5%) 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).
KW - Iron chelation therapy
KW - Transfusion medicine
KW - Transfusion-dependent anemias
UR - http://www.scopus.com/inward/record.url?scp=77950682176&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=77950682176&partnerID=8YFLogxK
U2 - 10.3324/haematol.2009.014696
DO - 10.3324/haematol.2009.014696
M3 - Article
C2 - 19951979
AN - SCOPUS:77950682176
VL - 95
SP - 557
EP - 566
JO - Haematologica
JF - Haematologica
SN - 0390-6078
IS - 4
ER -