Single-center retrospective study of the effectiveness and toxicity of the oral iron chelating drugs deferiprone and deferasirox

PloS One
Nancy OlivieriBrenda L Gallie

Abstract

Iron overload, resulting from blood transfusions in patients with chronic anemias, has historically been controlled with regular deferoxamine, but its parenteral requirement encouraged studies of orally-active agents, including deferasirox and deferiprone. Deferasirox, licensed by the US Food and Drug Administration in 2005 based upon the results of randomized controlled trials, is now first-line therapy worldwide. In contrast, early investigator-initiated trials of deferiprone were prematurely terminated after investigators raised safety concerns. The FDA declined market approval of deferiprone; years later, it licensed the drug as "last resort" therapy, to be prescribed only if first-line drugs had failed. We undertook to evaluate the long-term effectiveness and toxicities of deferiprone and deferasirox in one transfusion clinic. Under an IRB-approved study, we retrospectively inspected the electronic medical records of consented iron-loaded patients managed between 2009 and 2015 at The University Health Network (UHN), Toronto. We compared changes in liver and heart iron, adverse effects and other outcomes, in patients treated with deferiprone or deferasirox. Although deferiprone was unlicensed in Canada, one-third (n = 41) o...Continue Reading

References

Jun 24, 1976·The New England Journal of Medicine·R D PropperD G Nathan
Jul 1, 1989·Lancet·M G ZurloS Terzoli
Jun 20, 1985·The New England Journal of Medicine·L WolfeD G Nathan
Apr 6, 1995·The New England Journal of Medicine·N F OlivieriG Koren
Sep 1, 1994·The New England Journal of Medicine·G M BrittenhamJ W Harris
Sep 1, 1994·The New England Journal of Medicine·N F OlivieriA R Cohen
Aug 13, 1998·The New England Journal of Medicine·N F OlivieriK A Fleming
Nov 25, 1998·British Journal of Haematology·B WonkeA V Hoffbrand
Feb 26, 2000·British Journal of Haematology·A R CohenF Tricta
Aug 3, 2000·The New England Journal of Medicine·E AngelucciG Lucarelli
Oct 29, 2000·The Ceylon Medical Journal·G N LucasM Fernandopulle
Jan 13, 2001·Acta Haematologica·G C Del VecchioD De Mattia
Oct 18, 2001·Journal of Midwifery & Women's Health·J E Thompson
Jun 18, 2002·Blood Cells, Molecules & Diseases·Aurelio MaggioMassimo Midiri
Jul 9, 2002·British Journal of Haematology·Adriana CeciFernando Tricta
Oct 25, 2002·The New England Journal of Medicine·David G Nathan, David J Weatherall
Mar 29, 2003·The Ceylon Medical Journal·G N LucasI Weerasinghe
May 21, 2003·European Journal of Haematology·Ching-Tien PengChang-Hai Tsai
Jun 5, 2003·Hemoglobin·Massimo GaliaUNKNOWN Multicenter Trial Group of the Society for the Study of Thalassemia and Haemoglobinopathies
Jun 6, 2003·Blood·Gary M BrittenhamDavid J Weatherall
Jul 9, 2003·British Journal of Haematology·Pensri PootrakulA Victor Hoffbrand
Apr 15, 2004·Indian Journal of Pediatrics·V P ChoudhryA N Malaviya
May 19, 2004·British Journal of Haematology·Chaim HershkoGiuseppe Masera
Jun 26, 2004·Journal of Pediatric Hematology/oncology·Emanuela D'AngeloVittorio Carnelli
Jul 9, 2004·Radiotherapy and Oncology : Journal of the European Society for Therapeutic Radiology and Oncology·John M AkuduguRichard P Hill
Nov 27, 2004·Acta Haematologica·A TaherA V Hoffbrand
Dec 1, 2004·The Hematology Journal : the Official Journal of the European Haematology Association·Vasiliki AlymaraKonstantinos L Bourantas
Feb 3, 2005·CMAJ : Canadian Medical Association Journal = Journal De L'Association Medicale Canadienne·Edoardo G GianniniVincenzo Savarino
Mar 2, 2005·Best Practice & Research. Clinical Haematology·A Victor Hoffbrand
Jan 3, 2006·Blood Cells, Molecules & Diseases·Antonis KattamisChristos Kattamis

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Citations

Dec 15, 2019·Journal of Magnetic Resonance Imaging : JMRI·Pandji TriadyaksaPaul E Sijens
Feb 6, 2020·The Pharmacogenomics Journal·Jesús María Hernández SánchezAna Eugenia Rodríguez Vicente
Dec 18, 2020·British Journal of Clinical Pharmacology·Nicholas P GiangrecoNicholas P Tatonetti
Apr 21, 2021·Expert Opinion on Drug Safety·Laura GrechJoseph Borg
Jan 10, 2020·Journal of Medical Ethics·Arthur Schafer

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