Deferiprone (tradenames include Ferriprox) is a drug that chelates iron and is used to treat iron overload in thalassaemia major.[1] It was first approved for use in treating thalassaemia major in 1994[2] and had been licensed for use in Europe and Asia for many years while awaiting approval in Canada and the United States.[1] On October 14, 2011, it was approved for use in the US under the FDA's accelerated approval program.[3]
Controversy
Deferiprone was at the center of a protracted struggle between Nancy Olivieri, a Canadian haematologist and researcher, and the Hospital for Sick Children and the pharmaceutical company Apotex, that started in 1996 and delayed approval of the drug in North America.[4] Olivieri's data suggested deferiprone leads to progressive hepatic fibrosis.[5][6][7]
See also
Deferoxamine
Deferasirox
References
^ abSavulescu, J (2004). "Thalassaemia major: The murky story of deferiprone". BMJ. 328 (7436): 358–9. doi:10.1136/bmj.328.7436.358. PMC 341373. PMID 14962851.
^Staff, Cipla. Cipla's History Archived 2015-10-27 at the Wayback Machine
^FDA NEWS RELEASE: FDA Approves Ferripox (deferiprone) to Treat Patients with Excess Iron in the Body, Oct. 14, 2011 https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm275814.htm
^Viens, A M; Savulescu, J (2004). "Introduction to the Olivieri symposium". Journal of Medical Ethics. 30 (1): 1–7. doi:10.1136/jme.2003.006577. PMC 1757126. PMID 14872065.
^Brittenham, G. M; Nathan, D. G; Olivieri, N. F; Porter, J. B; Pippard, M; Vichinsky, E. P; Weatherall, D. J (2003). "Deferiprone and hepatic fibrosis". Blood. 101 (12): 5089–90, author reply 5090–1. doi:10.1182/blood-2002-10-3173. PMID 12788794.
^Wanless, I. R; Sweeney, G; Dhillon, A. P; Guido, M; Piga, A; Galanello, R; Gamberini, M. R; Schwartz, E; Cohen, A. R (2002). "Lack of progressive hepatic fibrosis during long-term therapy with deferiprone in subjects with transfusion-dependent beta-thalassemia". Blood. 100 (5): 1566–9. doi:10.1182/blood-2002-01-0306. PMID 12176871.
^Cribb, Robert (2019-02-27). "UHN patients given unlicensed drug that led to diabetes, liver dysfunction and one death, study finds". The Star. Toronto. Retrieved 2019-02-27.
5. 造血細胞移植後のサラセミア患者の長期マネージメント long term management of the thalassemic patient after hematopoietic cell transplantation
English Journal
Iron distribution and histopathological characterization of the liver and heart of β-thalassemic mice with parenteral iron overload: Effects of deferoxamine and deferiprone.
Experimental and toxicologic pathology : official journal of the Gesellschaft für Toxikologische Pathologie.Exp Toxicol Pathol.2014 Sep;66(7):333-43. doi: 10.1016/j.etp.2014.03.002. Epub 2014 Jun 3.
The liver and heart are the major target organs for iron accumulation and iron toxicity in β-thalassemia. To mimic the phenomenon of heavy iron overload resulting from repeated blood transfusions, a total of 180mg of iron dextran was intraperitoneally injected into C57BL/6J mice (WT) and heterozygo
A new deferiprone controlled release system obtained by ultrasound-assisted compression.
Aguilar-De-Leyva A1, Gonçalves-Araujo T, Daza V, Caraballo I.
Pharmaceutical development and technology.Pharm Dev Technol.2014 Sep;19(6):728-34. doi: 10.3109/10837450.2013.829091. Epub 2013 Aug 28.
OBJECTIVES: This study implements the design of an innovative dosage form using ultrasound-assisted compression of thermoplastic polymers and the development of controlled release tablets for the oral administration of deferiprone in two doses per day.METHODS: Binary matrix tablets containing deferi
Abstract Aims: The pathophysiological role of iron in Parkinson's disease (PD) was assessed by a chelation strategy aimed at reducing oxidative damage associated with regional iron deposition without affecting circulating metals. Translational cell and animal models provided concept proofs and a del
Ferriprox® (deferiprone) is an iron chelator indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. ... This website contains information for a ...
Deferiprone comes as a tablet to take by mouth. It is usually taken three times a day, in the morning, at mid-day, and in the evening. Deferiprone may be taken with or without food, but taking it with meals may help to prevent nausea ...