Iron Chelation Therapy in the Context of the Latest Iron Science

Abstract

Iron chelation therapy (ICT) has been available since 1963, when the current reference standard chelator deferoxamine (DFO) was introduced for the clinical treatment of iron overload. DFO, along with our improved knowledge of the management of iron overload, has since greatly reduced the morbidity of patients with thalassemia and markedly improved their prognosis, providing the potential for almost normal longevity. However, the rigorous and demanding DFO treatment regimen created the need for more convenient therapy options and led to the development of oral chelators. Deferiprone was the first oral iron chelator and was launched in the mid 1990s. Research and development has continued, aiming to identify an ICT that is effective, convenient, well tolerated, and has properties that will allow optimization of therapy, based on our increased understanding of iron overload and iron chelation. Deferasirox, the first once-daily oral chelator, is now available in many countries around the world.