Abstract

According to WHO data, approximately 56,000 babies are born each year with a major thalassaemia, including at least 30,000 who require lifelong transfusions to survive. Of those transfused, under 40% obtain adequate chelation therapy. An estimated 100,000 patients worldwide are currently living with regular transfusions, but at least 3,000 die each year in their teens or early 20s from uncontrolled iron overload, mainly due to heart failure. With no physiological excretory pathway, iron from transfused blood accumulates in the liver, heart, endocrine and other organs causing tissue damage and impairment in function. Chelating agents can remove the excess iron and prevent complications, the most serious of which is death from heart failure due to myocardial siderosis. Progressive cardiac iron loading eventually leads to left ventricular (LV) dilatation and reduction in LV ejection fraction but this is a late manifestation of severe myocardial siderosis and once clinical evidence of heart failure has developed, the prognosis is poor.