Thalassaemia major: the murky story of deferiprone

摘要

Every year over 200 000 babies are born with thalassaemia major. They have a life expectancy of less than 30 years and are dependent on blood transfusions. Repeated transfusions result in cirrhosis of the liver, cardiomyopathy, endocrinopathies, and death due to haemosiderosis. Desferoxamine, an iron chelator, has been used for more than 30 years to treat haemosiderosis. It is given by daily, subcutaneous, slow injection, with inconvenience and local reactions resulting in suboptimal compliance in about half the patients. Despite desferoxamine, cardiac disease is still responsible for 70% of all deaths in these patients. Developing an orally administered chelating agent has therefore been a major objective in the care of patients with thalassaemia. Unfortunately the development of such a drug (deferiprone) has resulted in one of the most acrimonious and destructive of conflicts between a clinical researcher (Nancy Olivieri) and a drug company (Apotex). This dispute raises several ethical issues, which are discussed in a symposium in the Journal of Medical Ethics and in part here.