Although
blood transfusions are important for patients with
anemia, chronic transfusions inevitably lead to
iron overload as humans cannot actively remove excess
iron. The cumulative effects of
iron overload lead to significant morbidity and mortality, if untreated. Although the current reference standard
iron chelator deferoxamine has been used clinically for over four decades, its effectiveness is limited by a demanding therapeutic regimen that leads to poor compliance.
Deferasirox (
Exjade(R),
ICL670, Novartis Pharma AG, Basel, Switzerland) is a once-daily, oral
iron chelator approved for the treatment of transfusional
iron overload in adult and pediatric patients. The efficacy and safety of
deferasirox have been established in a comprehensive clinical development program involving patients with various transfusion-dependent
anemias.
Deferasirox has a dose-dependent effect on
iron burden, and is as efficacious as
deferoxamine at comparable therapeutic doses.
Deferasirox therapy can be tailored to a patient's needs, as response is related to both dose and
iron intake. Since
deferasirox has a long half-life and is present in the plasma for 24 hours with once-daily dosing, it is unique in providing constant chelation coverage with a single dose. The availability of this convenient, effective, and well tolerated
therapy represents a significant advance in the management of transfusional
iron overload.