This review provides an update on advances in the area of iron chelation therapy, including new indications and uses of currently available agents, and preliminary data on potential new agents in development.
Two new oral agents, deferasirox and deferiprone, have become available in the last 8 years. These have been used at higher doses, in combination with the older agent desferrioxamine, and recent trials’ data have shown efficacy in preventing or treating the toxicity associated with iron overload. Advances in measuring tissue iron noninvasively by magnetic resonance techniques have enhanced diagnostic capabilities and allowed for more precise measurement and monitoring of iron burden. The primary use of chelation has been transfusional iron overload. There is now an increasing body of evidence for the benefits of iron chelation in myelodysplasia, pre-stem cell transplantation, and potentially in the treatment of malignancies. Two new iron chelators are in development, one in phase 3 clinical trials and the other in preliminary animal studies.
The last decade has ushered in a new era in iron chelation therapy. Coupled with advances in tissue iron quantitation, there is tremendous promise of an individually tailored approach to chelation, and subsequent reduction in morbidity and mortality.
Weill Cornell Medical College, New York, New York, USA
Correspondence to Sujit Sheth, MD, Professor of Clinical Pediatrics, Division of Pediatric Hematology and Oncology, Weill Cornell Medical College, 525 East 68th Street, P – 695, New York, NY 10065, USA. Tel: +1 212 746 3400; fax: +1 212 746 8609; e-mail: firstname.lastname@example.org