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Five-year trial of deferiprone chelation therapy in thalassaemia major patients

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dc.contributor.author Taher A.
dc.contributor.author Aoun E.
dc.contributor.author Sharara A.I.
dc.contributor.author Mourad F.
dc.contributor.author Gharzuddine W.
dc.contributor.author Koussa S.
dc.contributor.author Inati A.
dc.contributor.author Dhillon A.P.
dc.contributor.author Hoffbrand A.V.
dc.contributor.editor
dc.date Dec-2004
dc.date.accessioned 2017-10-05T15:39:12Z
dc.date.available 2017-10-05T15:39:12Z
dc.date.issued 2004
dc.identifier 10.1159/000081268
dc.identifier.isbn
dc.identifier.issn 00015792
dc.identifier.uri http://hdl.handle.net/10938/16729
dc.description.abstract Twelve thalassaemia major patients have been given deferiprone 75 mg-kg body weight daily as iron chelation therapy for 5 years. Their ages ranged from 18 to 34 years (mean 24.2) at the end of the study. Two patients were hepatitis C virus (HCV) mRNA positive and a further 5 were positive for HCV antibody. The mean serum ferritin level fell significantly from 4,302 ± 2,245 μg-l SD at baseline to 3,032 ± 1,155 μg-l at 2 years (p = 0.037) and 2,229 ± 1,070 μg-l (p = 0.007) at 5 years. At the end of the study, liver iron ranged from 3.59 to 23.7 mg-g dry weight (mean 11.9 ± 5.4), 3 patients having levels 15 mg-g. There was no significant change in serum AST levels, but ALT levels fell significantly at 2 years (p = 0.019) and 5 years (p = 0.001). Liver biopsy at the end of the study showed no evidence of hepatic fibrosis caused by deferiprone. Cardiac studies showed no overall change in left ventricular ejection fraction but a significant improvement in isovolumic relaxation time (p = 0.045). We conclude that in this albeit small group of thalassaemia major patients, deferiprone was a safe long-term method of iron chelation. In a minority, higher doses of deferiprone or a combination with desferrioxamine would be needed to lower liver iron below 15 mg-g. Copyright © 2004 S. Karger AG, Basel.
dc.format.extent
dc.format.extent Pages: (179-183)
dc.language English
dc.publisher BASEL
dc.relation.ispartof Publication Name: Acta Haematologica; Publication Year: 2004; Volume: 112; no. 4; Pages: (179-183);
dc.relation.ispartofseries
dc.relation.uri
dc.source Scopus
dc.subject.other
dc.title Five-year trial of deferiprone chelation therapy in thalassaemia major patients
dc.type Article
dc.contributor.affiliation Taher, A., Department of Internal Medicine, American University of Beirut, Medical Center, Beirut, Lebanon, Chronic Care Center, Hazmieh, Lebanon
dc.contributor.affiliation Aoun, E., Department of Internal Medicine, American University of Beirut, Medical Center, Beirut, Lebanon
dc.contributor.affiliation Sharara, A.I., Department of Internal Medicine, American University of Beirut, Medical Center, Beirut, Lebanon
dc.contributor.affiliation Mourad, F., Department of Internal Medicine, American University of Beirut, Medical Center, Beirut, Lebanon
dc.contributor.affiliation Gharzuddine, W., Department of Internal Medicine, American University of Beirut, Medical Center, Beirut, Lebanon
dc.contributor.affiliation Koussa, S., Chronic Care Center, Hazmieh, Lebanon
dc.contributor.affiliation Inati, A., Chronic Care Center, Hazmieh, Lebanon
dc.contributor.affiliation Dhillon, A.P., Roy. Free and Univ. Coll. Med. Sch., London, United Kingdom
dc.contributor.affiliation Hoffbrand, A.V., Roy. Free and Univ. Coll. Med. Sch., London, United Kingdom, Royal Free Hampstead NHS Trust, Pond Street, London NW3 2QG, United Kingdom
dc.contributor.authorAddress Hoffbrand, A.V.; Royal Free Hampstead NHS Trust, Pond Street, London NW3 2QG, United Kingdom; email: v.hoffbrand@rfc.ucl.ac.uk
dc.contributor.authorCorporate University: American University of Beirut Medical Center; Faculty: Faculty of Medicine; Department: Internal Medicine;
dc.contributor.authorDepartment Internal Medicine
dc.contributor.authorDivision
dc.contributor.authorEmail v.hoffbrand@rfc.ucl.ac.uk
dc.contributor.authorFaculty Faculty of Medicine
dc.contributor.authorInitials Taher, A
dc.contributor.authorInitials Aoun, E
dc.contributor.authorInitials Sharara, AI
dc.contributor.authorInitials Mourad, F
dc.contributor.authorInitials Gharzuddine, W
dc.contributor.authorInitials Koussa, S
dc.contributor.authorInitials Inati, A
dc.contributor.authorInitials Dhillon, AP
dc.contributor.authorInitials Hoffbrand, AV
dc.contributor.authorOrcidID
dc.contributor.authorReprintAddress Hoffbrand, AV (reprint author), UCL Royal Free Hampstead NHS Trust, Pond St, London NW3 2QG, England.
dc.contributor.authorResearcherID Dhillon, Amar-C-5619-2009
dc.contributor.authorUniversity American University of Beirut Medical Center
dc.description.cited Anderson LJ, 2002, LANCET, V360, P516, DOI 10.1016-S0140-6736(02)09740-4; Balfour JAB, 1999, DRUGS, V58, P553; BARRY M, 1971, LANCET, V1, P100; BRITTENHAM GM, 1994, NEW ENGL J MED, V331, P567, DOI 10.1056-NEJM199409013310902; Gabutti V, 1996, ACTA HAEMATOL-BASEL, V95, P26; Gharzuddine W S, 2002, Eur J Echocardiogr, V3, P47, DOI 10.1053-euje.2001.0114; Hoffbrand AV, 2003, BLOOD, V102, P17, DOI 10.1182-blood-2002-06-1867; ISHAK K, 1995, J HEPATOL, V22, P696, DOI 10.1016-0168-8278(95)80226-6; Mourad FH, 2003, BRIT J HAEMATOL, V121, P187, DOI 10.1046-j.1365-2141.2003.04240.x; Olivieri NF, 1998, NEW ENGL J MED, V339, P417, DOI 10.1056-NEJM199808133390701; Olivieri NF, 1997, BLOOD, V89, P739; Piga A, 2003, HAEMATOLOGICA, V88, P489; Porter JB, 2001, BRIT J HAEMATOL, V115, P239, DOI 10.1046-j.1365-2141.2001.03195.x; SCHEUER PJ, 2000, LIVER BIOPSY INTERPR, P274; Taher A, 1999, ACTA HAEMATOL-BASEL, V101, P173, DOI 10.1159-000040949; Tondury P, 1998, BRIT J HAEMATOL, V101, P413, DOI 10.1046-j.1365-2141.1998.00725.x; Wanless IR, 2002, BLOOD, V100, P1566, DOI 10.1182-blood-2002-01-0306; Wonke B, 1998, BRIT J HAEMATOL, V103, P361
dc.description.citedCount 11
dc.description.citedTotWOSCount 11
dc.description.citedWOSCount 9
dc.format.extentCount 5
dc.identifier.articleNo
dc.identifier.coden ACHAA
dc.identifier.pubmedID 15564727
dc.identifier.scopusID 9144230310
dc.identifier.url
dc.publisher.address ALLSCHWILERSTRASSE 10, CH-4009 BASEL, SWITZERLAND
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dc.relation.ispartofConferenceCode
dc.relation.ispartofConferenceDate
dc.relation.ispartofConferenceHosting
dc.relation.ispartofConferenceLoc
dc.relation.ispartofConferenceSponsor
dc.relation.ispartofConferenceTitle
dc.relation.ispartofFundingAgency
dc.relation.ispartOfISOAbbr Acta Haematol.
dc.relation.ispartOfIssue 4
dc.relation.ispartOfPart
dc.relation.ispartofPubTitle Acta Haematologica
dc.relation.ispartofPubTitleAbbr Acta Haematol.
dc.relation.ispartOfSpecialIssue
dc.relation.ispartOfSuppl
dc.relation.ispartOfVolume 112
dc.source.ID WOS:000225332800001
dc.type.publication Journal
dc.subject.otherAuthKeyword Deferiprone
dc.subject.otherAuthKeyword Echocardiography
dc.subject.otherAuthKeyword Iron chelation
dc.subject.otherAuthKeyword Liver biopsy
dc.subject.otherAuthKeyword Thalassemia
dc.subject.otherChemCAS alanine aminotransferase, 9000-86-6, 9014-30-6
dc.subject.otherChemCAS aspartate aminotransferase, 9000-97-9
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 Antibodies, Viral
dc.subject.otherChemCAS deferiprone, 30652-11-0
dc.subject.otherChemCAS Ferritins, 9007-73-2
dc.subject.otherChemCAS Iron Chelating Agents
dc.subject.otherChemCAS Iron, 7439-89-6
dc.subject.otherChemCAS Pyridones
dc.subject.otherChemCAS RNA, Viral
dc.subject.otherIndex alanine aminotransferase
dc.subject.otherIndex aspartate aminotransferase
dc.subject.otherIndex deferiprone
dc.subject.otherIndex ferritin
dc.subject.otherIndex hepatitis C antibody
dc.subject.otherIndex iron
dc.subject.otherIndex messenger RNA
dc.subject.otherIndex virus RNA
dc.subject.otherIndex adult
dc.subject.otherIndex age distribution
dc.subject.otherIndex alanine aminotransferase blood level
dc.subject.otherIndex article
dc.subject.otherIndex aspartate aminotransferase blood level
dc.subject.otherIndex body weight
dc.subject.otherIndex chelation therapy
dc.subject.otherIndex clinical article
dc.subject.otherIndex controlled study
dc.subject.otherIndex drug safety
dc.subject.otherIndex dry weight
dc.subject.otherIndex female
dc.subject.otherIndex ferritin blood level
dc.subject.otherIndex heart hemodynamics
dc.subject.otherIndex heart left ventricle ejection fraction
dc.subject.otherIndex Hepatitis C virus
dc.subject.otherIndex human
dc.subject.otherIndex human tissue
dc.subject.otherIndex iron chelation
dc.subject.otherIndex liver biopsy
dc.subject.otherIndex liver fibrosis
dc.subject.otherIndex liver weight
dc.subject.otherIndex male
dc.subject.otherIndex priority journal
dc.subject.otherIndex statistical significance
dc.subject.otherIndex thalassemia major
dc.subject.otherIndex Adolescent
dc.subject.otherIndex Adult
dc.subject.otherIndex Antibodies, Viral
dc.subject.otherIndex Enzyme Tests
dc.subject.otherIndex Female
dc.subject.otherIndex Ferritins
dc.subject.otherIndex Heart Function Tests
dc.subject.otherIndex Hepacivirus
dc.subject.otherIndex Humans
dc.subject.otherIndex Iron
dc.subject.otherIndex Iron Chelating Agents
dc.subject.otherIndex Liver
dc.subject.otherIndex Male
dc.subject.otherIndex Pyridones
dc.subject.otherIndex RNA, Viral
dc.subject.otherIndex Thalassemia
dc.subject.otherKeywordPlus IRON OVERLOAD
dc.subject.otherKeywordPlus BETA-THALASSEMIA
dc.subject.otherKeywordPlus LIVER-IRON
dc.subject.otherKeywordPlus DESFERRIOXAMINE
dc.subject.otherKeywordPlus DEFEROXAMINE
dc.subject.otherKeywordPlus EFFICACY
dc.subject.otherKeywordPlus FIBROSIS
dc.subject.otherWOS Hematology


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