Cited by 49Hemoglobinopathies and Related DisordersIron Metabolism and DisordersBiomedical Ethics and Regulation

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

Kontoghiorghes, George J.·2008·Hemoglobin

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.

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Crossref · 10.1080/03630260701726533

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  • Due to serious side effects, the applicability of iron chelators during cancer treatment is hampered.

    “Unfortunately, due to serious side effects in several cases, e.g., hearing abnormalities, nephropathy, optic neuropathy, and growth failure in children, their applicability during cancer treatment is hampered [135,139].”

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Authors
Kontoghiorghes, G. J.
Corresponding author
Kontoghiorghes, George J.
Venue
Hemoglobin
Published
2008-01
Volume
32
Issue
1-2
Pages
1-15
ISSN
  • 0363-0269 (ISSN-L)
  • 1532-432X
OpenAlex
W2013938992
Cited by
49
References
30
Citations by year
2026: 1 · 2025: 1 · 2024: 4 · 2022: 2 · 2021: 1 · 2020: 2 · 2019: 3 · 2018: 1 · 2017: 1 · 2016: 3 · 2015: 3 · 2014: 3 · 2013: 1
FWCI
3.34
Type
journal article
Language
en
Open access
closed
Locations
  • Hemoglobin (publishedVersion)
  • PubMed (publishedVersion)
Topics
  • Health Sciences › Medicine › Genetics › Hemoglobinopathies and Related Disorders
  • Health Sciences › Medicine › Hematology › Iron Metabolism and Disorders
  • Health Sciences › Medicine › Physiology › Biomedical Ethics and Regulation
Keywords
iron chelation therapy, deferasirox, deferiprone, deferoxamine, transfusional iron overload, thalassemia, cardiac iron, drug safety, drug efficacy, drug costs, accelerated approval, renal failure, liver failure, agranulocytosis, regulatory policy, pharmaceutical marketing
MeSH terms
Deferiprone, Deferasirox, Benzoates, Deferoxamine, Drug Therapy, Combination, Humans, Iron, Iron Chelating Agents, Pyridones, Thalassemia, Triazoles, Chelation Therapy, Siderophores, Drug Approval, Risk Assessment, Iron Overload

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