Beta-thalassaemia: emergence of new and improved iron chelators for treatment.
Beta-thalassaemia is an inherited blood disorder which through repeated blood transfusions and enhanced iron uptake from the gastrointestinal tract, results in marked iron overload. Untreated, the iron accumulation results in the dysfunction of vital organs such as the heart and liver. At present, the most effective treatment for beta-thalassaemia is the use of the iron chelator, desferrioxamine, which is expensive, orally inactive and requires long subcutaneous infusions. In this concise review, we will focus on novel chelators which show therapeutic potential to replace desferrioxamine. Furthermore, we will discuss the potential of combined iron chelation therapy and the principle that, in the future, the use of more than just one chelator may be beneficial in tailoring individual iron chelation regimens.
Wong
Cynthia
C
The Iron Metabolism and Chelation Program, Children's Cancer Institute Australia for Medical Research, PO Box 81, High Street, Randwick, New South Wales Sydney 2031, Australia.
Richardson
Des R
DR
D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D019190:Iron Overload / Q000209:etiology; D017086:beta-Thalassemia / Q000150:complications / Q000188:drug therapy* / Q000235:genetics
D007502:Iron Chelating Agents