With three iron chelating agents now available, management options have substantially increased
Outcomes in patients with thalassaemia major have been revolutionised over the past 50 years. Without transfusions, death usually occurred in the first decade of life. In the 1950s, transfusions were given to manage the symptoms of anaemia, which resulted in increased survival but significant morbidity. In the 1960s, regular blood transfusions were introduced to maintain relatively high mean haemoglobin levels in order to suppress the production of abnormal red cells in the bone marrow. This permitted good quality of life in childhood, but led to cardiac death from transfusional iron overload at a mean age of 18 years.1 Fortunately, the parenteral iron chelating agent desferrioxamine was also introduced in the 1960s, and its use to control iron load led to improved survival2 and reduced morbidity.3 Nevertheless, the difficult treatment regimen (subcutaneous infusion for 8–12 hours per night, 3–7 nights per week) resulted in poor compliance. Cardiomyopathy remains the most common cause of premature death in patients with thalassaemia,3 even in well chelated patients.4
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Vasili Berdoukas is a consultant for ApoPharma, the company that markets deferiprone, and he has a confidentiality agreement with Novartis for the development of deferasirox. He has also received travel assistance and honoraria for attending conferences and for presentations at such conferences.