beta-thalassaemia has served as a paradigm for chelation management for over three decades, both in terms of defining the complications of transfusional
iron overload, and demonstrating the benefits of
chelation therapy.
Iron chelation therapy can be used to reduce unacceptably high tissue
iron levels, or to maintain current levels if these are deemed safe, by matching the rate of transfused
iron.
Chelation therapy should be tailored to the individual patient, based on the transfusional
iron loading rate and the current level of
iron load both intra- and extra-hepatically, for example in the myocardium. In general, it is preferable to prevent extra-hepatic complications by controlling the body
iron load rather than attempting to rescue patients once extra-hepatic complications have developed.
Deferoxamine, which has been available since the late 1970s and is given parenterally, has been shown to prolong life and decrease morbidity from
iron overload in patients who comply with
therapy.
Deferiprone may control body
iron as oral monotherapy in a variable proportion of patients but is now more frequently used in combinations with
deferoxamine, either to control total levels of body
iron or to reduce increased levels of myocardial
iron. In this article, recent advances in the use of
deferasirox, a once-daily oral
iron chelator, are reviewed. Large-scale prospective trials show efficacy with an acceptable safety profile in adults and children with up to 5 years follow-up. Recent evidence suggests that
deferasirox up to 30 mg/kg/day can be safely administered to patients with serum
ferritin levels between 500 and 1000 mg/L, while doses above 30 mg/kg/day can be given to patients with substantial
iron overload or with high transfusion rates. Further, prospective data show that myocardial
iron can be effectively decreased with this chelation treatment.