title: Five-year trial of deferiprone chelation therapy in thalassaemia major patients.
abstract: Twelve thalassaemia major patients have been given deferiprone 75 mg/kg body weight daily as iron chelation therapy for 5 years. Their ages ranged from 18 to 34 years (mean 24.2) at the end of the study. Two patients were hepatitis C virus (HCV) mRNA positive and a further 5 were positive for HCV antibody. The mean serum ferritin level fell significantly from 4,302 +/- 2,245 microg/l SD at baseline to 3,032 +/- 1,155 microg/l at 2 years (p = 0.037) and 2,229 +/- 1,070 microg/l (p = 0.007) at 5 years. At the end of the study, liver iron ranged from 3.59 to 23.7 mg/g dry weight (mean 11.9 +/- 5.4), 3 patients having levels >15 mg/g. There was no significant change in serum AST levels, but ALT levels fell significantly at 2 years (p = 0.019) and 5 years (p = 0.001). Liver biopsy at the end of the study showed no evidence of hepatic fibrosis caused by deferiprone. Cardiac studies showed no overall change in left ventricular ejection fraction but a significant improvement in isovolumic relaxation time (p = 0.045). We conclude that in this albeit small group of thalassaemia major patients, deferiprone was a safe long-term method of iron chelation. In a minority, higher doses of deferiprone or a combination with desferrioxamine would be needed to lower liver iron below 15 mg/g.
authors.affiliation: Department of Internal Medicine, American University of Beirut, Medical Center, Beirut, Lebanon.
authors.forename: A
authors.initials: A
authors.lastname: Taher
authors.forename: E
authors.initials: E
authors.lastname: Aoun
authors.forename: A I
authors.initials: AI
authors.lastname: Sharara
authors.forename: F
authors.initials: F
authors.lastname: Mourad
authors.forename: W
authors.initials: W
authors.lastname: Gharzuddine
authors.forename: S
authors.initials: S
authors.lastname: Koussa
authors.forename: A
authors.initials: A
authors.lastname: Inati
authors.forename: A P
authors.initials: AP
authors.lastname: Dhillon
authors.forename: A V
authors.initials: AV
authors.lastname: Hoffbrand
mesh_terms: D000293:Adolescent; D000328:Adult; D000914:Antibodies, Viral / Q000097:blood; D004796:Clinical Enzyme Tests; D000077543:Deferiprone; D005260:Female; D005293:Ferritins / Q000097:blood; D006334:Heart Function Tests; D016174:Hepacivirus / Q000235:genetics / Q000276:immunology; D006801:Humans; D007501:Iron / Q000032:analysis; D007502:Iron Chelating Agents / Q000008:administration & dosage*; D008099:Liver / Q000737:chemistry; D008297:Male; D011728:Pyridones / Q000008:administration & dosage*; D012367:RNA, Viral / Q000097:blood; D013789:Thalassemia / Q000188:drug therapy*
chemical_list: D000914:Antibodies, Viral; D007502:Iron Chelating Agents; D011728:Pyridones; D012367:RNA, Viral; D000077543:Deferiprone; D005293:Ferritins; D007501:Iron