Thalassaemia major: the murky story of deferiprone

BMJ 2004; 328 doi: https://doi.org/10.1136/bmj.328.7436.358 (Published 12 February 2004) Cite this as: BMJ 2004;328:358
  1. Julian Savulescu, Uehiro chair in practical ethics (julian.savulescu@philosophy.oxford.ac.uk)
  1. Oxford Uehiro Centre for Practical Ethics, University of Oxford, Oxford OX1 1PT

    Conducting life saving research properly and quickly is a moral imperative

    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.1 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).2 w1-w4 Thisdispute raises several ethical issues, which are discussed in a symposium in the Journal of Medical Ethics3 and in part here.

    Dr Olivieri began to study deferiprone in 1989. In 1993 Apotex began to provide financial support for her trials. By March 1996 Dr …

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