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.

Abstract

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.

• Authors: George J Kontoghiorghes
• Journal: Hemoglobin (Hemoglobin) Vol. 32 Issue 1-2 Pg. 1-15 ( 2008) ISSN: 0363-0269 [Print] England
• PMID: 18274978 (Publication Type: Journal Article, Review)

Chemical References

• Benzoates
• Iron Chelating Agents
• Pyridones
• Siderophores
• Triazoles
• Deferiprone
• Iron
• Deferoxamine
• Deferasirox

Topics

• Benzoates (adverse effects, chemistry, economics, therapeutic use)
• Chelation Therapy (economics, ethics)
• Deferasirox
• Deferiprone
• Deferoxamine (chemistry, economics, therapeutic use)
• Drug Approval
• Drug Therapy, Combination
• Humans
• Iron (metabolism)
• Iron Chelating Agents (adverse effects, chemistry, economics, therapeutic use)
• Iron Overload (drug therapy)
• Pyridones (chemistry, economics, therapeutic use)
• Risk Assessment
• Siderophores (chemistry, economics, therapeutic use)
• Thalassemia (drug therapy, epidemiology)
• Triazoles (adverse effects, chemistry, economics, therapeutic use)