@article{3001684,
    title = "Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload",
    author = "Aydinok, Y. and Kattamis, A. and Cappellini, M.D. and El-Beshlawy, A. and Origa, R. and Elalfy, M. and Kilinç, Y. and Perrotta, S. and Karakas, Z. and Viprakasit, V. and Habr, D. and Constantinovici, N. and Shen, J. and Porter, J.B. and HYPERION Investigators",
    journal = "Blood advances",
    year = "2015",
    volume = "125",
    number = "25",
    pages = "3868-3877",
    publisher = "American Society of Hematology",
    doi = "10.1182/blood-2014-07-586677",
    keywords = "creatinine;  deferasirox;  deferoxamine;  ferritin;  iron;  benzoic acid derivative;  deferasirox;  deferoxamine;  iron chelating agent;  siderophore;  triazole derivative, abdominal pain;  adult;  arthritis;  Article;  central nervous system infection;  child;  creatinine blood level;  diarrhea;  disease control;  disease severity;  DRESS syndrome;  drug effect;  drug safety;  drug withdrawal;  female;  ferritin blood level;  fever;  geometry;  heart arrhythmia;  heart function;  heart hemosiderosis;  heart left ventricle ejection fraction;  human;  iron overload;  liver hemosiderosis;  major clinical study;  male;  minimum inhibitory concentration;  monotherapy;  multicenter study;  nausea;  open study;  phase 2 clinical trial;  priority journal;  prospective study;  pruritus;  teratoma;  adolescent;  adverse effects;  blood transfusion;  chemistry;  clinical trial;  drug effects;  heart;  heart muscle;  iron overload;  liver;  young adult, Adolescent;  Adult;  Benzoates;  Blood Transfusion;  Child;  Deferoxamine;  Female;  Heart;  Humans;  Iron Chelating Agents;  Iron Overload;  Liver;  Male;  Myocardium;  Siderophores;  Triazoles;  Young Adult",
    abstract = "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 (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∗<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 www.clinicaltrials.gov as #NCT01254227. © 2015 by The American Society of Hematology."
}