Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload

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

Research output: Contribution to journalArticle

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. 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∗

Original languageEnglish
Pages (from-to)3868-3877
Number of pages10
JournalBlood
Volume125
Issue number25
DOIs
Publication statusPublished - Jun 18 2015

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

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    Aydinok, Y., Kattamis, A., Cappellini, M. D., El-Beshlawy, A., Origa, R., Elalfy, M., Kilinç, Y., Perrotta, S., Karakas, Z., Viprakasit, V., Habr, D., Constantinovici, N., Shen, J., & Porter, J. B. (2015). Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload. Blood, 125(25), 3868-3877. https://doi.org/10.1182/blood-2014-07-586677