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Vol. 112, No. 4, 2004
Issue release date: November 2004
Acta Haematol 2004;112:179–183

Five-Year Trial of Deferiprone Chelation Therapy in Thalassaemia major Patients

Taher A. · Aoun E. · Sharara A.I. · Mourad F. · Gharzuddine W. · Koussa S. · Inati A. · Dhillon A.P. · Hoffbrand A.V.
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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 µg/l SD at baseline to 3,032 ± 1,155 µg/l at 2 years (p = 0.037) and 2,229 ± 1,070 µg/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.

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