The New England journal of medicine
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Clinical Trial Controlled Clinical Trial
Long-term safety and effectiveness of iron-chelation therapy with deferiprone for thalassemia major.
Deferiprone is an orally active iron-chelating agent that is being evaluated as a treatment for iron overload in thalassemia major. Studies in an animal model showed that prolonged treatment is associated with a decline in the effectiveness of deferiprone and exacerbation of hepatic fibrosis. ⋯ Deferiprone does not adequately control body iron burden in patients with thalassemia and may worsen hepatic fibrosis.