@article {107855,
	title = {Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload.},
	journal = {Blood},
	volume = {125},
	year = {2015},
	month = {2015 Jun 18},
	pages = {3868-77},
	abstract = {<p>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-\&lt;10 ms; left ventricular ejection fraction [LVEF] >=56\%) followed by optional switch to DFX monotherapy when achieving mT2* \&gt;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* \&lt;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 <a href="http://www.clinicaltrials.gov" title="www.clinicaltrials.gov">www.clinicaltrials.gov</a> as $\#$NCT01254227.</p>
},
	keywords = {Adolescent, Adult, Benzoates, Blood Transfusion, Child, Deferoxamine, Female, Heart, Humans, Iron Chelating Agents, Iron Overload, Liver, Male, Myocardium, Siderophores, Triazoles, Young Adult},
	issn = {1528-0020},
	doi = {10.1182/blood-2014-07-586677},
	author = {Yesim Aydinok and Antonis Kattamis and Cappellini, M Domenica and Amal El-Beshlawy and Origa, Raffaella and Mohsen Elalfy and Kilin{\c c}, Yurdanur and Perrotta, Silverio and Karakas, Zeynep and Vip Viprakasit and Dany Habr and Constantinovici, Niculae and Shen, Junwu and John B. Porter}
}
