Iron loading and its clinical implications

The main aspects of iron loading and the consequent clinical implications described in this series of articles are summarized in this final chapter. Despite mechanisms to maintain iron homeostasis, harmful iron accumulation can occur in patients with hereditary defects of regulatory proteins, such as hepcidin, or with transfusion-dependent anemias, such as thalassemia and myelodysplastic syndromes. Identifying the role of nontransferrin bound iron in the pathogenesis of disease allows for better treatment strategies to prevent and reverse iron toxicity. In addition, accurate noninvasive methods to reliably assess iron accumulation and chelation are now available. Continuous chelation coverage, which can be achieved with combination therapy (deferoxamine and deferiprone) or deferasirox, is expected to provide optimal protection from iron toxicity. As more long-term data on these drugs accumulate, the role of oral and combination chelation therapies in relation to blood transfusion, as well as other iron overload disorders, will become clearer.