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

This analysis evaluated the effects of deferasirox on liver iron concentration in moderate and heavily iron-overloaded patients with β-thalassaemia from the ESCALATOR trial (n=231). Mean liver iron concentrations (LIC) decreased significantly from 21.1±8.2 to 14.2±12.1mg Fe/g dry weight (dw) at 2yr (P<0.001) in patients with LIC ≥7mg Fe/g dw at baseline; patients with LIC <7mg Fe/g dw maintained these levels over the treatment period. The proportion of patients with LIC <7mg 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 <7mg Fe/g dw at a mean dose of 22.4±5.2mg/kg/d and significantly reducing LIC in patients with LIC ≥7mg Fe/g dw at a mean dose of 25.7±4.2mg/kg/d, along with a manageable safety profile.