TY - JOUR
TI - Randomized controlled trial of deferiprone or deferoxamine in
beta-thalassemia major patients with asymptomatic myocardial siderosis
AU - Pennell, DJ
AU - Berdoukas, V
AU - Karagiorga, M
AU - Ladis, V
AU - Piga, A
AU - and Aessopos, A
AU - Gotsis, ED
AU - Tanner, MA
AU - Smith, GC and
AU - Westwood, MA
AU - Wonke, B
AU - Galanello, R
JO - Blood advances
PY - 2006
VL - 107
TODO - 9
SP - 3738-3744
PB - AMER SOC HEMATOLOGY
SN - null
TODO - 10.1182/blood-2005-07-2948
TODO - null
TODO - 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 mu g/L vs -466
mu g/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.
ER -