Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis.
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 end point 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/d and deferoxamine was 43 mg/kg for 5.7 d/wk. Compliance was 94% +/- 5.3% and 93% +/- 9.7% (P = .81), respectively. The improvement in myocardial T2(*) was significantly greater for deferiprone than deferoxamine (27% vs 13%; P = .023). Left ventricular ejection fraction increased significantly more in the deferiprone-treated group (3.1% vs 0.3% absolute units; P = .003). The changes in liver iron level (-0.93 mg/g dry weight vs -1.54 mg/g dry weight; P = .40) and serum ferritin level (-181 microg/L vs -466 microg/L; P = .16), respectively, were not significantly different between groups. The most frequent adverse events were transient gastrointestinal symptoms for deferiprone-treated 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.
Pennell
Dudley J
DJ
Royal Brompton Hospital, Sydney Street, London SW3 6NP, United Kingdom. d.pennell@ic.ac.uk
Berdoukas
Vasili
V
Karagiorga
Markissia
M
Ladis
Vasili
V
Piga
Antonio
A
Aessopos
Athanassios
A
Gotsis
Efstathios D
ED
Tanner
Mark A
MA
Smith
Gill C
GC
Westwood
Mark A
MA
Wonke
Beatrix
B
Galanello
Renzo
R
D000328:Adult; D009202:Cardiomyopathies / Q000188:drug therapy* / Q000209:etiology* / Q000378:metabolism; D000077543:Deferiprone; D003676:Deferoxamine / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D011728:Pyridones / Q000009:adverse effects / Q000627:therapeutic use*; D012806:Siderosis / Q000188:drug therapy* / Q000209:etiology* / Q000378:metabolism; D017086:beta-Thalassemia / Q000150:complications* / Q000188:drug therapy* / Q000378:metabolism
D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D007501:Iron; D003676:Deferoxamine