Since 1962,
desferrioxamine (
deferoxamine, DFO) has been utilized for the treatment of secondary
hemosiderosis. For about 30 years, DFO
therapy has been performed as nightly continuous
subcutaneous infusion. About 20 years ago, the first oral
iron chelator (
deferiprone,
DFP) was presented. Concerns about potential side effects were responsible for the late acceptance and license of this
drug which is limited to the use as second-line
therapy for patients with
thalassemia major. During recent years,
chelation therapy and its evaluation started to progress rapidly. Clinical research and
drug development as well as the introduction of new methods for the assessment of
iron overload contributed to these advances. By using cardiac T2 (*) MRI it was possible to examine the specific effect of a
chelator on myocardial
siderosis. Clinical studies using this method indicated superiority of
DFP compared to DFO with respect to the treatment of myocardial
siderosis. Several retrospective and first prospective clinical trials seem to confirm this observation. In parallel, treatment strategies based on the combination of DFO and
DFP have been developed. Using both drugs simultaneously or sequentially, additive and synergistic effects contribute to the fast elimination of
iron from different organs at risk for siderotic damage.
Deferasirox (DSX) is a recently developed oral
chelator which shows good efficacy and tolerability in patients with transfusional
hemosiderosis due to various underlying disorders. Long-term studies will define the future importance of DSX for
iron chelation treatment. For the first time, there is a choice between three commercially available
chelating agents for patients with transfusional
iron overload. This will allow a highly effective, individually tailored treatment hopefully leading to a fundamental improvement of patients' life expectancy and quality.