Abstract:
Following 1-yr deferasirox therapy in the ESCALATOR study, 57percent 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; 67.4percent (P0.0001) achieved treatment success. By the end of the extension, 66.2percent 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; P0.001) and median serum ferritin by 929ng-mL (baseline 3356; P0.0001). There was a concomitant improvement in liver function markers (P0.0001). Fewer drug-related adverse events were reported in extension than core study (23.8percent vs. 44.3percent). 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. © 2011 John Wiley and Sons A-S.