TY - JOUR
T1 - Importance of optimal dosing ≥30mg/kg/d during deferasirox treatment
T2 - 2.7-yr follow-up from the ESCALATOR study in patients with β-thalassaemia
AU - Taher, Ali
AU - Elalfy, Mohsen S.
AU - Al Zir, Kusai
AU - Daar, Shahina
AU - Al Jefri, Abdullah
AU - Habr, Dany
AU - Kriemler-Krahn, Ulrike
AU - El-Ali, Ali
AU - Roubert, Bernard
AU - El-Beshlawy, Amal
PY - 2011/10
Y1 - 2011/10
N2 - Following 1-yr deferasirox therapy in the ESCALATOR study, 57% of previously chelated patients with β-thalassaemia achieved treatment success (maintenance of or reduction in liver iron concentration (LIC) vs. baseline LIC). Seventy-eight per cent had dose increases at median of 26wk, suggesting that 1-yr results may not have reflected full deferasirox efficacy. Extension data are presented here. Deferasirox starting dose was 20mg/kg/d (increases to 30/40mg/kg/d permitted in the core/extension, respectively). Efficacy was primarily assessed by absolute change in LIC and serum ferritin. Overall, 231 patients received deferasirox in the extension; 67.4% (P<0.0001) achieved treatment success. By the end of the extension, 66.2% of patients were receiving doses ≥30mg/kg/d. By the end of the 1-yr extension, mean LIC had decreased by 6.6±9.4mg Fe/g dw (baseline 19.6±9.2; P<0.001) and median serum ferritin by 929ng/mL (baseline 3356; P<0.0001). There was a concomitant improvement in liver function markers (P<0.0001). Fewer drug-related adverse events were reported in extension than core study (23.8% vs. 44.3%). Doses ≥30mg/kg/d were generally required because of high transfusional iron intake and high baseline serum ferritin levels, highlighting the importance of administering an adequate dose to achieve net negative iron balance.
AB - Following 1-yr deferasirox therapy in the ESCALATOR study, 57% of previously chelated patients with β-thalassaemia achieved treatment success (maintenance of or reduction in liver iron concentration (LIC) vs. baseline LIC). Seventy-eight per cent had dose increases at median of 26wk, suggesting that 1-yr results may not have reflected full deferasirox efficacy. Extension data are presented here. Deferasirox starting dose was 20mg/kg/d (increases to 30/40mg/kg/d permitted in the core/extension, respectively). Efficacy was primarily assessed by absolute change in LIC and serum ferritin. Overall, 231 patients received deferasirox in the extension; 67.4% (P<0.0001) achieved treatment success. By the end of the extension, 66.2% of patients were receiving doses ≥30mg/kg/d. By the end of the 1-yr extension, mean LIC had decreased by 6.6±9.4mg Fe/g dw (baseline 19.6±9.2; P<0.001) and median serum ferritin by 929ng/mL (baseline 3356; P<0.0001). There was a concomitant improvement in liver function markers (P<0.0001). Fewer drug-related adverse events were reported in extension than core study (23.8% vs. 44.3%). Doses ≥30mg/kg/d were generally required because of high transfusional iron intake and high baseline serum ferritin levels, highlighting the importance of administering an adequate dose to achieve net negative iron balance.
KW - Deferasirox
KW - Efficacy
KW - Iron chelation therapy
KW - Iron overload
KW - β-thalassaemia
UR - http://www.scopus.com/inward/record.url?scp=80053251950&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=80053251950&partnerID=8YFLogxK
U2 - 10.1111/j.1600-0609.2011.01662.x
DO - 10.1111/j.1600-0609.2011.01662.x
M3 - Article
C2 - 21668502
AN - SCOPUS:80053251950
SN - 0902-4441
VL - 87
SP - 355
EP - 365
JO - European Journal of Haematology
JF - European Journal of Haematology
IS - 4
ER -