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Title: Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload
Authors: Yesim Aydinok
Antonis Kattamis
M. Domenica Cappellini
Amal El-Beshlawy
Raffaella Origa
Mohsen Elalfy
Yurdanur Kilinç
Silverio Perrotta
Zeynep Karakas
Vip Viprakasit
Dany Habr
Niculae Constantinovici
Junwu Shen
John B. Porter
Ege University Medical School
University of Athens
Universita degli Studi di Milano
Cairo University
Universita degli Studi di Cagliari
Ain Shams University
Cukurova Universitesi
Child and of General and Specialist Surgery
Istanbul Tip Fakultesi
Mahidol University
Novartis Pharmaceuticals
Novartis International AG
UCL Cancer Institute
Keywords: Biochemistry, Genetics and Molecular Biology;Immunology and Microbiology;Medicine
Issue Date: 18-Jun-2015
Citation: Blood. Vol.125, No.25 (2015), 3868-3877
Abstract: © 2015 by The American Society of Hematology. 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; left ventricular ejection fraction [LVEF] ≥56%) followed by optional switch to DFX monotherapy when achieving mT2∗>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 (Gmean<inf>month12/24</inf>/Gmean<inf>baseline</inf>) 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∗<5 ms and 1 died (suspected central nervous system infection). Safety was consistent with established monotherapies. Results show clinically meaningful improvements in mT2∗in about one-third of patients remaining on treatment at month 24, alongside rapid decreases in LIC in this heavily iron-overloaded, difficult-to-treat population. Combination therapy may be useful when rapid LIC reduction is required, regardless of myocardial iron overload. This trial was registered at as #NCT01254227.
ISSN: 15280020
Appears in Collections:Scopus 2011-2015

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