|Drug class||Iron chelator|
|Main uses||Iron overload in thalassaemia major|
|Side effects||Red-brown urine, nausea, abdominal pain, vomiting|
|Typical dose||25 mg/kg TID|
|Elimination half-life||2 to 3 hours|
|Excretion||Kidney (75 to 90% in 24 hours)|
|Chemical and physical data|
|Molar mass||139.154 g·mol−1|
|3D model (JSmol)|
|(what is this?)|
Deferiprone, sold under the brand name Ferriprox among others, is a medication used to treat iron overload due to blood transfusions in thalassaemia major. It is taken by mouth. Evidence supports improved blood markers but not symptoms or survival.
Common side effects include red-brown urine, nausea, abdominal pain, and vomiting. Other side effects may include low white blood cells, infection, and prolonged QT. Use in pregnancy may harm the baby. It is an iron chelator.
Deferiprone became commercially available in 1994. It was initially approved in Europe and Asia. It is available as a generic medication. This was followed by approval in the United States in 2011. In the United Kingdom 100 tablets of 500 mg costs the NHS about £130. In the United States this amount costs about 7,500 USD.
Deferiprone in combination with another chelator is indicated in the European Union in those with thalassaemia major when monotherapy with any iron chelator is ineffective, or when prevention or treatment of life-threatening consequences of iron overload (mainly cardiac overload) justifies rapid or intensive correction.
Deferiprone was approved for medical use in the European Union in August 1999.
The safety and effectiveness of deferiprone is based on an analysis of data from twelve clinical studies in 236 participants. Participants in the study did not respond to prior iron chelation therapy. Deferiprone was considered a successful treatment for participants who experienced at least a 20 percent decrease in serum ferritin, a protein that stores iron in the body for later use. Half of the participants in the study experienced at least a 20 percent decrease in ferritin levels.
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. Olivieri's data suggested deferiprone leads to progressive hepatic fibrosis.
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