Iron Overload and Iron Chelation Therapy in Pediatric Patients

Abstract

Iron overload is an unfortunate clinical consequence of repeated blood transfusions that can cause significant organ damage, morbidity, and mortality in the absence of proper treatment. Pediatric patients with transfusion-dependent pathologies face the additional risk of growth failure and poor sexual development owing to iron build-up in the anterior pituitary gland. Iron chelation therapy is necessary for the removal of excess iron, but treatment efficacy and success are highly dependent on patient compliance. Deferoxamine is a well-established but inconvenient therapy requiring parenteral administration over extended periods of time. Patient compliance can be improved with use of the oral iron chelators deferasirox and deferiprone. Long-term data have shown deferasirox to have a good safety and efficacy profile in pediatric patients. KeywordsIron overload, hemoglobinopathies, pediatric, iron chelator, compliance Disclosure: Elliott Vichinsky, MD, is an investigator for and has received honoraria from Novartis.