Achieving treatment goals of reducing or maintaining body iron burden with deferasirox in patients with beta-thalassaemia: results from the ESCALATOR study.

摘要

This analysis evaluated the effects of deferasirox on liver iron concentration in moderate and heavily iron-overloaded patients with beta-thalassaemia from the ESCALATOR trial (n = 231). Mean liver iron concentrations (LIC) decreased significantly from 21.1 +/- 8.2 to 14.2 +/- 12.1 mg Fe/g dry weight (dw) at 2 yr (P < 0.001) in patients with LIC >/= 7 mg Fe/g dw at baseline; patients with LIC < 7 mg Fe/g dw maintained these levels over the treatment period. The proportion of patients with LIC < 7 mg Fe/g dw increased from 9.4% at core baseline to 39.3% by the end of year 2. The results showed that deferasirox enabled therapeutic goals to be achieved, by maintaining LIC in patients with LIC < 7 mg Fe/g dw at a mean dose of 22.4 +/- 5.2 mg/kg/d and significantly reducing LIC in patients with LIC >/= 7 mg Fe/g dw at a mean dose of 25.7 +/- 4.2 mg/kg/d, along with a manageable safety profile.