The 18th ICOC Proceedings in Athens, Greece: New breakthrough in thalassemia leading to the complete treatment of iron overload and to hundreds of patients achieving and maintaining normal body iron stores. Ethical questions on chelation therapy.

A new era in thalassemia and other transfusional iron loading conditions was highlighted during the 18th International Conference on Chelation (ICOC) with reports that all excess iron accumulated from transfusions could be removed using the ICOC combination protocol of deferiprone (L1) (80-100 mg/kg/day) and subcutaneous deferoxamine (DFO) (40-60 mg/kg/day, at least 3 days per week), and that normal range body iron store levels (NRBISL) could be maintained using L1 monotherapy. Hundreds of patients in Cyprus, Greece, Italy, UK and elsewhere, maintain NRBISL, some for more than 9 years, and without complications. This gold standard of complete iron overload treatment is likely to change current practices, aims and protocols because it could prevent and also reverse cardiac, liver, endocrine and other organ complications as well as the incidence of infections and hepatocellular carcinomas. The overall morbidity and mortality in thalassemia and other transfusional iron loading conditions is expected to be substantially reduced. New applications of chelating drugs include renal, neurodegenerative, infectious diseases and ischemia reperfusion injury patients. Ethical questions have been raised on the role of pharmaceutical companies, the clinicians and the Hippocratic oath in relation to chelation therapy.

Ethical issues and risk/benefit assessment of iron chelation therapy: advances with deferiprone/deferoxamine combinations and concerns about the safety, efficacy and costs of deferasirox.

New developments in the area of iron and other metal metabolism and toxicity and the effects and uses of chelators have been presented at the 16th International Conference on Chelation (ICOC), Limassol, Cyprus in October 2006. Marketing practices by pharmaceutical companies, contradictory policies by regulatory authorities and ineffective policies by health authorities deprive thousands of thalassemia and other transfused patients of life saving iron chelating drugs and of efficacious chelation treatments. Thousands of patients were using deferasirox (DFRA) worldwide a few months after the European Union (EU) authorities, and about 1 year after the Food and Drugs Administration (FDA), proceeded to its accelerated approval with no sufficient evidence that the drug was efficacious, especially for clearing excess cardiac iron, and also safe. Cases of fatal, acute, irreversible renal and liver failure, fatal agranulocytosis and other toxicities have recently been reported with DFRA. The FDA has not yet approved deferiprone (L1) depriving thousands of patients of potentially life saving treatment. The high cost of DFRA at 60 euros/g, L1 at 5.5 euros/g and deferoxamine (DFO) at 8.3 euros/g, diminishes the prospects of universal chelation therapy, especially for patients in developing countries. The safety and efficacy record of L1, DFO, and their combination in particular, appear to provide universal solutions in the treatment of transfusional iron overload, and also in reducing mortality because of their ability to clear rapidly and effectively excess cardiac iron.