TY - JOUR
T1 - Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload
AU - Aydinok, Yesim
AU - Kattamis, Antonis
AU - Cappellini, M. Domenica
AU - El-Beshlawy, Amal
AU - Origa, Raffaella
AU - Elalfy, Mohsen
AU - Kilinç, Yurdanur
AU - Perrotta, Silverio
AU - Karakas, Zeynep
AU - Viprakasit, Vip
AU - Habr, Dany
AU - Constantinovici, Niculae
AU - Shen, Junwu
AU - Porter, John B.
PY - 2015/6/18
Y1 - 2015/6/18
N2 - Deferasirox (DFX) monotherapy is effective for reducing myocardial and liver iron concentrations (LIC), although some patients may require intensive chelation for a limited duration. HYPERION, an open-label single-arm prospective phase 2 study, evaluated combination DFX-deferoxamine (DFO) in patients with severe transfusional myocardial siderosis (myocardial [m] T2∗5-10 ms. Mean dose was 30.5 mg/kg per day DFX and 36.3 mg/kg per day DFO on a 5-day regimen. Geometric mean mT2∗ratios (Gmeanmonth12/24/Gmeanbaseline) were 1.09 and 1.30, respectively, increasing from 7.2 ms at baseline (n = 60) to 7.7 ms at 12 (n = 52) and 9.5 ms at 24 months (n = 36). Patients (17 of 60; 28.3%) achieved mT2∗≥10 ms and ≥10% increase from baseline at month 24; 15 switched to monotherapy during the study based on favorable mT2∗. LIC decreased substantially from a baseline of 33.4 to 12.8 mg Fe/g dry weight at month 24 (-52%). LVEF remained stable with no new arrhythmias/cardiac failure. Five patients discontinued with mT2∗
AB - Deferasirox (DFX) monotherapy is effective for reducing myocardial and liver iron concentrations (LIC), although some patients may require intensive chelation for a limited duration. HYPERION, an open-label single-arm prospective phase 2 study, evaluated combination DFX-deferoxamine (DFO) in patients with severe transfusional myocardial siderosis (myocardial [m] T2∗5-10 ms. Mean dose was 30.5 mg/kg per day DFX and 36.3 mg/kg per day DFO on a 5-day regimen. Geometric mean mT2∗ratios (Gmeanmonth12/24/Gmeanbaseline) were 1.09 and 1.30, respectively, increasing from 7.2 ms at baseline (n = 60) to 7.7 ms at 12 (n = 52) and 9.5 ms at 24 months (n = 36). Patients (17 of 60; 28.3%) achieved mT2∗≥10 ms and ≥10% increase from baseline at month 24; 15 switched to monotherapy during the study based on favorable mT2∗. LIC decreased substantially from a baseline of 33.4 to 12.8 mg Fe/g dry weight at month 24 (-52%). LVEF remained stable with no new arrhythmias/cardiac failure. Five patients discontinued with mT2∗
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U2 - 10.1182/blood-2014-07-586677
DO - 10.1182/blood-2014-07-586677
M3 - Article
C2 - 25934475
AN - SCOPUS:84935029971
VL - 125
SP - 3868
EP - 3877
JO - Blood
JF - Blood
SN - 0006-4971
IS - 25
ER -