Deferasirox is an orally active chelator, highly selective for iron (III). It binds iron in a 2:1 ratio and promotes excretion of iron, primarily in the faeces.
Exjade has been studied in 411 adult and 292 paediatric patients with chronic iron overload due to blood transfusions.
Doses of 20 and 30mg/kg daily in adults and paediatric patients, respectively, with beta thalassaemia receiving frequent transfusions led to reductions in liver iron concentration and serum ferritin. At these doses the ratios of iron excretion : iron intake showed a net iron balance in adult patients and net iron removal in paediatric patients. Similar responses were demonstrated in iron-overloaded patients with other anaemias.
Serum ferritin levels, assessed monthly, reflected changes in liver concentration indicating that serum ferritin levels can be used to monitor therapy response.
In a comparative study of deferasirox to desferrioxamine in over 500 patients suffering from thalassaemia and transfusional iron overload, deferasirox failed to demonstrate non-inferiority criteria in the total population. However, post-hoc analysis demonstrated non-inferiority in a subgroup of patients with liver iron concentration >7mg Fe/g dw.
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