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Patients with chronic anaemias such as thalassaemia, sickle cell disease, congenital rare anaemias and myelodysplastic syndromes require regular blood transfusions in order to improve both quality of life and survival. Humans are unable to eliminate the iron released from the breakdown of transfused red blood cells and the excess iron is deposited as haemosiderin and ferritin in the liver, spleen, endocrine organs and myocardium. The accumulation of toxic quantities of iron causes tissue damage and leads to complications such as heart failure, diabetes, hypothyroidism and liver failure. Morbidity and mortality in regularly transfused thalassaemia patients are due primarily to the effects of iron overload rather than to the underlying disease.
Iron chelators mobilize tissue iron by forming soluble, stable complexes that are then excreted in the faeces and/or urine. Although desferrioxamine has been available for the past 40 years, its poor oral bioavailability and short plasma half-life necessitate parenteral administration and prolonged infusions. Further, therapy was expensive and cumbersome, and led to significant non-compliance.
The introduction of the world's first oral iron chelator, deferiprone, by Cipla in 1995 revolutionised the treatment of iron overload, and brought the benefits of oral iron chelation therapy within reach of thousands of patients globally.
DESIROX ( Deferasirox) is yet another breakthrough in oral iron chelation therapy. Its once-daily dosing and availability as a dispersible tablet are unique features, and represent an important advance in therapy.