Long-term administration of desferrioxamine in thalassaemia major

Abstract

thalassaemia major. Studies of desferrioxamine (DFA) as a specific iron-chelating agent in the treatment of haemosiderosis have been reported in various conditions, but there has been only limited evaluation of its long-term use in thalassaemia major. In the present study 1 g DFA was given intramuscularly daily 6 days per week in 3 patients for 5 to 8 years, and in another 7 patients for 10 to 22 months. In addition, 500 mg DFA was added to each unit of donor blood at the time of transfusions. Urinary iron excretion after DFA was estimated using atomic absorption spectro-photometry both before and periodically during DFA treatment. During follow-up, cardiac and liver functions were assessed by ECG, chest x-ray, and liver function tests. The results show that DFA therapy increased urinary iron excretion, and that this was associated with a significant improvement in the clinical symptoms and signs of haemosiderosis and with return towards normal in the ECG and liver function tests. The majority of children with thalassaemia major who do not succumb to the complications associated with severe anaemia nor to postsplenectomy infection usually die during the second or the third decade from intractable cardiac failure, liver cirrhosis, or diabetes mellitus. The pathogenesis of these complications is based on an excessive deposition of iron in vital organs which is associated with or followed by a steadily progressive fibrosi