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Tailoring iron chelation by iron intake and serum ferritin: The prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias

Show simple item record Cappellini M.D. Porter J. El-Beshlawy A. Li C.-K. Seymour J.F. Elalfy M. Gattermann N. Giraudier S. Lee J.-W. Chan L.L. Lin K.-H. Rose C. Taher A. Thein S.L. Viprakasit V. Habr D. Domokos G. Roubert B. Kattamis A.
dc.contributor.editor Apr-2010 2017-10-05T16:01:06Z 2017-10-05T16:01:06Z 2010
dc.identifier 10.3324/haematol.2009.014696
dc.identifier.issn 03906078
dc.description.abstract Background: Following a clinical evaluation of deferasirox (Exjade®) it was concluded that, in addition to baseline body iron burden, ongoing transfusional iron intake should be considered when selecting doses. The 1-year EPIC study, the largest ever investigation conducted for an iron chelator, is the first to evaluate whether fixed starting doses of deferasirox, based on transfusional iron intake, with dose titration guided by serum ferritin trends and safety markers, provides clinically acceptable chelation in patients (aged ≥2 years) with transfusional hemosiderosis from various types of anemia. Design and Methods: The recommended initial dose was 20 mg-kg-day for patients receiving 2-4 packed red blood cell units-month and 10 or 30 mg-kg-day was recommended for patients receiving less or more frequent transfusions, respectively. Dose adjustments were based on 3-month serum ferritin trends and continuous assessment of safety markers. The primary efficacy end-point was change in serum ferritin after 52 weeks compared with baseline. Results: The 1744 patients enrolled had the following conditions; thalassemia (n=1115), myelodysplastic syndromes (n=341), aplastic anemia (n=116), sickle cell disease (n=80), rare anemias (n=43) and other transfused anemias (n=49). Overall, there was a significant reduction in serum ferritin from baseline (-264 ng-mL; P0.0001), reflecting dosage adjustments and ongoing iron intake. The most common (5percent) adverse events were gastrointestinal disturbances (28percent) and skin rash (10percent). Conclusions: Analysis of this large, prospectively collected data set confirms the response to chelation ther- apy across various anemias, supporting initial deferasirox doses based on transfusional iron intake, with subsequent dose titration guided by trends in serum ferritin and safety markers ( identifier: NCT00171821). © 2010 Ferrata Storti Foundation.
dc.format.extent Pages: (557-566)
dc.language English
dc.publisher PAVIA
dc.relation.ispartof Publication Name: Haematologica; Publication Year: 2010; Volume: 95; no. 4; Pages: (557-566);
dc.source Scopus
dc.title Tailoring iron chelation by iron intake and serum ferritin: The prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias
dc.type Article
dc.contributor.affiliation Cappellini, M.D., Università di Milano, Policlinico Foundation IRCCS, Milan, Italy
dc.contributor.affiliation Porter, J., University College London, London, United Kingdom
dc.contributor.affiliation El-Beshlawy, A., Cairo University, Cairo, Egypt
dc.contributor.affiliation Li, C.-K., Prince of Wales Hospital, Chinese University of Hong Kong, Hong Kong
dc.contributor.affiliation Seymour, J.F., Peter MacCallum Cancer Centre, Melbourne, Australia
dc.contributor.affiliation Elalfy, M., Ain Shams University, Cairo, Egypt
dc.contributor.affiliation Gattermann, N., Heinrich-Heine-University, Düsseldorf, Germany
dc.contributor.affiliation Giraudier, S., Hôpital Henri Mondor, Créteil, France
dc.contributor.affiliation Lee, J.-W., The Catholic University of Korea, Seoul, South Korea
dc.contributor.affiliation Chan, L.L., University Malaya Medical Centre, Kuala Lumpur, Malaysia
dc.contributor.affiliation Lin, K.-H., National Taiwan University Hospital, Taipei, Taiwan
dc.contributor.affiliation Rose, C., Hôpital Saint-Vincent de Paul (Groupe Francophone des Myélodysplasies), Lille, France
dc.contributor.affiliation Taher, A., American University of Beirut, Beirut, Lebanon
dc.contributor.affiliation Thein, S.L., King's College London School of Medicine, King's College Hospital, London, United Kingdom
dc.contributor.affiliation Viprakasit, V., Siriraj Hospital, Mahidol University, Prannok, Bangkoknoi, Bangkok, Thailand
dc.contributor.affiliation Habr, D., Novartis Pharmaceuticals Corp., East Hanover, NJ, United States
dc.contributor.affiliation Domokos, G., Novartis Pharma AG, Basel, Switzerland
dc.contributor.affiliation Roubert, B., Novartis Pharma AG, Basel, Switzerland
dc.contributor.affiliation Kattamis, A., First Department of Pediatrics, University of Athens, Athens, Greece
dc.contributor.authorAddress Cappellini, M. D.; Università di Milano, Policlinico Foundation IRCCS, Milan, Italy; email:
dc.contributor.authorCorporate University: American University of Beirut Medical Center; Faculty: Faculty of Medicine; Department: Pediatrics and Adolescent Medicine;
dc.contributor.authorDepartment Pediatrics and Adolescent Medicine
dc.contributor.faculty Faculty of Medicine
dc.contributor.authorInitials Cappellini, MD
dc.contributor.authorInitials Porter, J
dc.contributor.authorInitials El-Beshlawy, A
dc.contributor.authorInitials Li, CK
dc.contributor.authorInitials Seymour, JF
dc.contributor.authorInitials Elalfy, M
dc.contributor.authorInitials Gattermann, N
dc.contributor.authorInitials Giraudier, S
dc.contributor.authorInitials Lee, JW
dc.contributor.authorInitials Chan, LL
dc.contributor.authorInitials Lin, KH
dc.contributor.authorInitials Rose, C
dc.contributor.authorInitials Taher, A
dc.contributor.authorInitials Thein, SL
dc.contributor.authorInitials Viprakasit, V
dc.contributor.authorInitials Habr, D
dc.contributor.authorInitials Domokos, G
dc.contributor.authorInitials Roubert, B
dc.contributor.authorInitials Kattamis, A
dc.contributor.authorReprintAddress Cappellini, MD (reprint author), Univ Milan, Policlin Fdn IRCCS, Milan, Italy.
dc.contributor.authorResearcherID Hematologico, Cancer-E-3038-2012; cruces, bio-F-2549-2012; Li, Chi Kong-J-8456-2013
dc.contributor.authorUniversity American University of Beirut Medical Center
dc.description.cited ALREFAIE FN, 1995, BRIT J HAEMATOL, V91, P224, DOI 10.1111-j.1365-2141.1995.tb05274.x; Borgna-Pignatti C, 2004, HAEMATOLOGICA, V89, P1187; BRITTENHAM GM, 1993, AM J HEMATOL, V42, P81, DOI 10.1002-ajh.2830420116; Cappellini MD, 2008, BLOOD, V112; Cappellini MD, 2006, BLOOD, V107, P3455, DOI 10.1182-blood-2005-08-3430; Cohen AR, 2008, BLOOD, V111, P583, DOI 10.1182-blood-2007-08-109306; Galanello R, 2006, HAEMATOL-HEMATOL J, V91, P1343; Jensen PD, 1996, BRIT J HAEMATOL, V94, P288, DOI 10.1046-j.1365-2141.1996.d01-1795.x; Jensen PD, 2004, BRIT J HAEMATOL, V124, P697, DOI 10.1111-j.1365-2141.2004.04838.x; Kersten MJ, 1996, ANN HEMATOL, V73, P247, DOI 10.1007-s002770050236; Leitch HA, 2007, LEUKEMIA RES, V31, pS7, DOI 10.1016-S0145-2126(07)70460-5; List A.E, 2008, BLOOD, V112; Novartis Pharmaceuticals Corporation, 2009, EXJ DEF PRESCR INF; Olivieri NF, 1999, NEW ENGL J MED, V341, P99, DOI 10.1056-NEJM199907083410207; OLIVIERI NF, 1995, NEW ENGL J MED, V332, P918, DOI 10.1056-NEJM199504063321404; Piga A, 2006, HAEMATOL-HEMATOL J, V91, P873; Piga A, 2005, BLOOD, V106; Piperno A, 1998, HAEMATOLOGICA, V83, P447; PORTER JB, 1987, ACTA HAEMATOL-BASEL, V78, P198; Porter JB, 2001, BRIT J HAEMATOL, V115, P239, DOI 10.1046-j.1365-2141.2001.03195.x; Porter J, 2008, EUR J HAEMATOL, V80, P168, DOI 10.1111-j.1600-0609.2007.00985.x; Rose C, 2007, BLOOD, V110; Sickle Cell Society, 2008, STAND CLIN CAR AD SI; Taher A, 2009, BRIT J HAEMATOL, V147, P752, DOI 10.1111-j.1365-2141.2009.07908.x; Taher A, 2009, HAEMATOL-HEMATOL J, V94, P82; Valent P, 2008, EUR J CLIN INVEST, V38, P143, DOI 10.1111-j.1365-2362.2007.01915.x; Vichinsky E, 2007, BRIT J HAEMATOL, V136, P501, DOI 10.1111-j.1365-2141.2006.06455.x
dc.description.citedCount 110
dc.description.citedTotWOSCount 108
dc.description.citedWOSCount 104
dc.format.extentCount 10
dc.identifier.coden HAEMA
dc.identifier.pubmedID 19951979
dc.identifier.scopusID 77950682176
dc.publisher.address VIA GIUSEPPE BELLI 4, 27100 PAVIA, ITALY
dc.relation.ispartOfISOAbbr Haematol-Hematol. J.
dc.relation.ispartOfIssue 4
dc.relation.ispartofPubTitle Haematologica
dc.relation.ispartofPubTitleAbbr Haematologica
dc.relation.ispartOfVolume 95
dc.source.ID WOS:000277144800008
dc.type.publication Journal
dc.subject.otherAuthKeyword Iron chelation therapy
dc.subject.otherAuthKeyword Transfusion medicine
dc.subject.otherAuthKeyword Transfusion-dependent anemias
dc.subject.otherChemCAS alanine aminotransferase, 9000-86-6, 9014-30-6
dc.subject.otherChemCAS deferasirox, 201530-41-8
dc.subject.otherChemCAS deferiprone, 30652-11-0
dc.subject.otherChemCAS ferritin, 9007-73-2
dc.subject.otherChemCAS iron, 14093-02-8, 53858-86-9, 7439-89-6
dc.subject.otherChemCAS Benzoic Acids
dc.subject.otherChemCAS Ferritins, 9007-73-2
dc.subject.otherChemCAS Iron Chelating Agents
dc.subject.otherChemCAS Iron, Dietary
dc.subject.otherChemCAS Triazoles
dc.subject.otherChemCAS deferasirox
dc.subject.otherIndex alanine aminotransferase
dc.subject.otherIndex deferasirox
dc.subject.otherIndex deferiprone
dc.subject.otherIndex ferritin
dc.subject.otherIndex iron
dc.subject.otherIndex abdominal pain
dc.subject.otherIndex abnormal laboratory result
dc.subject.otherIndex acute kidney failure
dc.subject.otherIndex adolescent
dc.subject.otherIndex adult
dc.subject.otherIndex aged
dc.subject.otherIndex alanine aminotransferase blood level
dc.subject.otherIndex anemia
dc.subject.otherIndex aplastic anemia
dc.subject.otherIndex arthralgia
dc.subject.otherIndex article
dc.subject.otherIndex cataract
dc.subject.otherIndex child
dc.subject.otherIndex clinical trial
dc.subject.otherIndex controlled clinical trial
dc.subject.otherIndex controlled study
dc.subject.otherIndex diarrhea
dc.subject.otherIndex drug dose increase
dc.subject.otherIndex drug dose reduction
dc.subject.otherIndex drug dose titration
dc.subject.otherIndex drug efficacy
dc.subject.otherIndex drug eruption
dc.subject.otherIndex drug response
dc.subject.otherIndex drug safety
dc.subject.otherIndex drug withdrawal
dc.subject.otherIndex erythrocyte transfusion
dc.subject.otherIndex female
dc.subject.otherIndex ferritin blood level
dc.subject.otherIndex gastrointestinal symptom
dc.subject.otherIndex hemosiderosis
dc.subject.otherIndex human
dc.subject.otherIndex iron chelation
dc.subject.otherIndex iron intake
dc.subject.otherIndex major clinical study
dc.subject.otherIndex male
dc.subject.otherIndex multicenter study
dc.subject.otherIndex myelodysplastic syndrome
dc.subject.otherIndex nausea
dc.subject.otherIndex outcome assessment
dc.subject.otherIndex phase 3 clinical trial
dc.subject.otherIndex proteinuria
dc.subject.otherIndex recommended drug dose
dc.subject.otherIndex sickle cell anemia
dc.subject.otherIndex side effect
dc.subject.otherIndex thalassemia
dc.subject.otherIndex treatment duration
dc.subject.otherIndex upper abdominal pain
dc.subject.otherIndex vomiting
dc.subject.otherIndex Adolescent
dc.subject.otherIndex Adult
dc.subject.otherIndex Aged
dc.subject.otherIndex Aged, 80 and over
dc.subject.otherIndex Anemia
dc.subject.otherIndex Benzoic Acids
dc.subject.otherIndex Blood Transfusion
dc.subject.otherIndex Child
dc.subject.otherIndex Child, Preschool
dc.subject.otherIndex Female
dc.subject.otherIndex Ferritins
dc.subject.otherIndex Humans
dc.subject.otherIndex Iron Chelating Agents
dc.subject.otherIndex Iron Overload
dc.subject.otherIndex Iron, Dietary
dc.subject.otherIndex Male
dc.subject.otherIndex Middle Aged
dc.subject.otherIndex Prospective Studies
dc.subject.otherIndex Thalassemia
dc.subject.otherIndex Tissue Distribution
dc.subject.otherIndex Triazoles
dc.subject.otherIndex Young Adult
dc.subject.otherKeywordPlus BETA-THALASSEMIA MAJOR
dc.subject.otherKeywordPlus MYELODYSPLASTIC SYNDROMES
dc.subject.otherKeywordPlus DEFERIPRONE L1
dc.subject.otherKeywordPlus OVERLOAD
dc.subject.otherKeywordPlus THERAPY
dc.subject.otherKeywordPlus DEFEROXAMINE
dc.subject.otherKeywordPlus ICL670
dc.subject.otherKeywordPlus MANAGEMENT
dc.subject.otherKeywordPlus DIAGNOSIS
dc.subject.otherKeywordPlus TRIAL
dc.subject.otherWOS Hematology

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