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Blood, 1 May 2006, Vol. 107, No. 9, pp. 3738-3744.
Prepublished online as a Blood First Edition Paper on December 13, 2005; DOI 10.1182/blood-2005-07-2948.


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Submitted July 22, 2005
Accepted December 1, 2005

Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis

Dudley J Pennell*, Vasili Berdoukas, Markissia Karagiorga, Vasili Ladis, Antonio Piga, Athanassios Aessopos, Efstathios D Gotsis, Mark A Tanner, Gill C Smith, Mark A Westwood, Beatrix Wonke, and Renzo Galanello

National Heart and Lung Institute, Imperial College, London, United Kingdom; Royal Brompton Hospital, London, United Kingdom
Aghia Sophia Children's Hospital, Athens, Greece
Laikon Hospital, University of Athens, Athens, Greece
Institute Euromedica-Encephalos, Athens, Greece
Whittington Hospital, London, United Kingdom
Royal Brompton Hospital, London, United Kingdom
Ospedale Regionale per le Microcitemie, Cagliari, Italy

* Corresponding author; email: d.pennell{at}ic.ac.uk.

Most deaths in beta-thalassemia major result from cardiac complications due to iron overload. Differential effects on myocardial siderosis may exist between different chelators. A randomized controlled trial was performed in 61 patients previously maintained on subcutaneous deferoxamine. The primary endpoint was the change in myocardial siderosis (myocardial T2*) over 1 year in patients maintained on subcutaneous deferoxamine or those switched to oral deferiprone monotherapy. The dose of deferiprone was 92 mg/kg/day and deferoxamine was 43 mg/kg for 5.7 days/week. Compliance was 94 ± 5.3% and 93 ± 9.7% (p=0.81) respectively. The improvement in myocardial T2* was significantly greater for deferiprone than deferoxamine (27% vs 13%, p=0.023). Left ventricular ejection fraction increased significantly more in the deferiprone group (3.1% vs 0.3% absolute units, p=0.0034). The changes in liver iron (-0.93 vs -1.54mg/g dw, p=0.40), and serum ferritin (-181 vs -466ug/L, p=0.16) respectively were not significantly different between groups. The most frequent adverse events were transient gastrointestinal symptoms for deferiprone patients, and local reactions at the infusion site for deferoxamine. There were no episodes of agranulocytosis. Deferiprone monotherapy was significantly more effective than deferoxamine over 1 year in improving asymptomatic myocardial siderosis in beta-thalassemia major.


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