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Efficacy and safety of deferasirox, an oral iron chelator, in heavily iron-overloaded patients with β-thalassaemia: The ESCALATOR study

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dc.contributor.author Taher A.
dc.contributor.author El-Beshlawy A.
dc.contributor.author Elalfy M.S.
dc.contributor.author Al Zir K.
dc.contributor.author Daar S.
dc.contributor.author Habr D.
dc.contributor.author Kriemler-Krahn U.
dc.contributor.author Hmissi A.
dc.contributor.author Al Jefri A.
dc.contributor.editor
dc.date Jun-2009
dc.date.accessioned 2017-10-05T15:41:02Z
dc.date.available 2017-10-05T15:41:02Z
dc.date.issued 2009
dc.identifier 10.1111/j.1600-0609.2009.01228.x
dc.identifier.isbn
dc.identifier.issn 09024441
dc.identifier.uri http://hdl.handle.net/10938/17780
dc.description.abstract Objective: Many patients with transfusional iron overload are at risk for progressive organ dysfunction and early death and poor compliance with older chelation therapies is believed to be a major contributing factor. Phase II-III studies have shown that oral deferasirox 20-30 mg-kg-d reduces iron burden, depending on transfusional iron intake. Methods: The prospective, open-label, 1-yr ESCALATOR study in the Middle East was designed to evaluate once-daily deferasirox in patients ≥2 yr with β-thalassaemia major and iron overload who were previously chelated with deferoxamine and-or deferiprone. Most patients began treatment with deferasirox 20 mg-kg-d; doses were adjusted in response to markers of over- or under-chelation. The primary endpoint was treatment success, defined as a reduction in liver iron concentration (LIC) of ≥3 mg Fe-g dry weight (dw) if baseline LIC was ≥10 mg Fe-g dw, or final LIC of 1-7 mg Fe-g dw for patients with baseline LIC of 2 to 10 mg Fe-g dw. Results: Overall, 233-237 enrolled patients completed 1 yr's treatment. Mean baseline LIC was 18.0 ± 9.1 mg Fe-g dw, while median serum ferritin was 3356 ng-mL. After 1 yr's deferasirox treatment, the intent-to-treat population experienced a significant treatment success rate of 57.0percent (P = 0.016) and a mean reduction in LIC of 3.4 mg Fe-g dw. Changes in serum ferritin appeared to parallel dose increases at around 24 wk. Most patients (78.1percent) underwent dose increases above 20 mg-kg-d, primarily to 30 mg-kg-d. Drug-related adverse events were mostly mild to moderate and resolved without discontinuing treatment. Conclusions: The results of the ESCALATOR study in primarily heavily iron-overloaded patients confirm previous observations in patients with β-thalassaemia, highlighting the importance of timely deferasirox dose adjustments based on serum ferritin levels and transfusional iron intake to ensure patients achieve their therapeutic goal of maintenance or reduction in iron burden. © 2009 Blackwell Munksgaard.
dc.format.extent
dc.format.extent Pages: (458-465)
dc.language English
dc.publisher HOBOKEN
dc.relation.ispartof Publication Name: European Journal of Haematology; Publication Year: 2009; Volume: 82; no. 6; Pages: (458-465);
dc.relation.ispartofseries
dc.relation.uri
dc.source Scopus
dc.subject.other
dc.title Efficacy and safety of deferasirox, an oral iron chelator, in heavily iron-overloaded patients with β-thalassaemia: The ESCALATOR study
dc.type Article
dc.contributor.affiliation Taher, A., Department of Internal Medicine, Hematology, Oncology Division, American University of Beirut Medical Center, Beirut 1107 2020, Lebanon, American University, Beirut, Lebanon
dc.contributor.affiliation El-Beshlawy, A., Cairo University, Cairo, Egypt
dc.contributor.affiliation Elalfy, M.S., Ain Shams University, Cairo, Egypt
dc.contributor.affiliation Al Zir, K., National Thalassemia Center, Damascus, Syrian Arab Republic
dc.contributor.affiliation Daar, S., Sultan Qaboos University, Muscat, Oman
dc.contributor.affiliation Habr, D., Novartis Pharmaceuticals Corporation, NJ, United States
dc.contributor.affiliation Kriemler-Krahn, U., Novartis Pharma AG, Basel, Switzerland
dc.contributor.affiliation Hmissi, A., Novartis Pharma AG, Basel, Switzerland
dc.contributor.affiliation Al Jefri, A., King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia
dc.contributor.authorAddress Taher, A.; Department of Internal Medicine, Hematology, Oncology Division, American University of Beirut Medical Center, Beirut 1107 2020, Lebanon; email: ataher@aub.edu.lb
dc.contributor.authorCorporate University: American University of Beirut Medical Center; Faculty: Faculty of Medicine; Department: Internal Medicine; Division: Hematology-Oncology;
dc.contributor.authorDepartment Internal Medicine
dc.contributor.authorDivision Hematology-Oncology
dc.contributor.authorEmail ataher@aub.edu.lb
dc.contributor.authorFaculty Faculty of Medicine
dc.contributor.authorInitials Taher, A
dc.contributor.authorInitials El-Beshlawy, A
dc.contributor.authorInitials Elalfy, MS
dc.contributor.authorInitials Al Zir, K
dc.contributor.authorInitials Daar, S
dc.contributor.authorInitials Habr, D
dc.contributor.authorInitials Kriemler-Krahn, U
dc.contributor.authorInitials Hmissi, A
dc.contributor.authorInitials Al Jefri, A
dc.contributor.authorOrcidID
dc.contributor.authorReprintAddress Taher, A (reprint author), Amer Univ Beirut, Div Oncol, Dept Internal Med, Med Ctr, Beirut 11072020, Lebanon.
dc.contributor.authorResearcherID
dc.contributor.authorUniversity American University of Beirut Medical Center
dc.description.cited BRITTENHAM GM, 1994, NEW ENGL J MED, V331, P567, DOI 10.1056-NEJM199409013310902; Brunt EM, 2000, HEPATOLOGY, V31, P241, DOI 10.1002-hep.510310136; 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; Cunningham MJ, 2004, BLOOD, V104, P34, DOI 10.1182-blood-2003-09-3167; ELEFTHERIOU P, 2006, HAEMATOLOGICA S1, V91; Gabutti V, 1996, ACTA HAEMATOL-BASEL, V95, P26; Galanello R, 2006, HAEMATOL-HEMATOL J, V91, P1343; ISHAK K, 1995, J HEPATOL, V22, P696, DOI 10.1016-0168-8278(95)80226-6; KUCZMARSKI RJ, 2000, ADV DATA, V1, P27; Low Louis C. K., 2005, Indian Journal of Pediatrics, V72, P159, DOI 10.1007-BF02760702; Modell B, 2000, LANCET, V355, P2051, DOI 10.1016-S0140-6736(00)02357-6; Olivieri NF, 1999, NEW ENGL J MED, V341, P99, DOI 10.1056-NEJM199907083410207; OLIVIERI NF, 1994, NEW ENGL J MED, V331, P574, DOI 10.1056-NEJM199409013310903; Olivieri NF, 1997, BLOOD, V89, P739; Piga A, 2006, HAEMATOL-HEMATOL J, V91, P873; Piperno A, 1998, HAEMATOLOGICA, V83, P447; Porter J, 2004, BLOOD, V104, p872A; Roth C, 1997, EUR J PEDIATR, V156, P777, DOI 10.1007-s004310050711; VICHINSKY E, 2006, BLOOD, V106
dc.description.citedCount 55
dc.description.citedTotWOSCount 55
dc.description.citedWOSCount 55
dc.format.extentCount 8
dc.identifier.articleNo
dc.identifier.coden EJHAE
dc.identifier.pubmedID 19187278
dc.identifier.scopusID 65349152022
dc.identifier.url
dc.publisher.address 111 RIVER ST, HOBOKEN 07030-5774, NJ USA
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dc.relation.ispartofConferenceDate
dc.relation.ispartofConferenceHosting
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dc.relation.ispartofConferenceSponsor
dc.relation.ispartofConferenceTitle
dc.relation.ispartofFundingAgency
dc.relation.ispartOfISOAbbr Eur. J. Haematol.
dc.relation.ispartOfIssue 6
dc.relation.ispartOfPart
dc.relation.ispartofPubTitle European Journal of Haematology
dc.relation.ispartofPubTitleAbbr Eur. J. Haematol.
dc.relation.ispartOfSpecialIssue
dc.relation.ispartOfSuppl
dc.relation.ispartOfVolume 82
dc.source.ID WOS:000265408600007
dc.type.publication Journal
dc.subject.otherAuthKeyword β-thalassaemia
dc.subject.otherAuthKeyword Deferasirox
dc.subject.otherAuthKeyword Iron chelation
dc.subject.otherAuthKeyword Transfusional iron overload
dc.subject.otherChemCAS deferasirox, 201530-41-8
dc.subject.otherChemCAS ferritin, 9007-73-2
dc.subject.otherChemCAS Benzoic Acids
dc.subject.otherChemCAS Deferoxamine, 70-51-9
dc.subject.otherChemCAS Ferritins, 9007-73-2
dc.subject.otherChemCAS Iron, 7439-89-6
dc.subject.otherChemCAS Iron Chelating Agents
dc.subject.otherChemCAS Pyridones
dc.subject.otherChemCAS Triazoles
dc.subject.otherChemCAS deferasirox
dc.subject.otherChemCAS deferiprone, 30652-11-0
dc.subject.otherIndex deferasirox
dc.subject.otherIndex ferritin
dc.subject.otherIndex abdominal pain
dc.subject.otherIndex adolescent
dc.subject.otherIndex adult
dc.subject.otherIndex article
dc.subject.otherIndex beta thalassemia
dc.subject.otherIndex child
dc.subject.otherIndex child development
dc.subject.otherIndex child growth
dc.subject.otherIndex cholecystitis
dc.subject.otherIndex cholelithiasis
dc.subject.otherIndex controlled study
dc.subject.otherIndex creatinine blood level
dc.subject.otherIndex diarrhea
dc.subject.otherIndex dose response
dc.subject.otherIndex drug dose increase
dc.subject.otherIndex drug efficacy
dc.subject.otherIndex drug eruption
dc.subject.otherIndex drug safety
dc.subject.otherIndex female
dc.subject.otherIndex ferritin blood level
dc.subject.otherIndex fever
dc.subject.otherIndex heart failure
dc.subject.otherIndex heart function
dc.subject.otherIndex human
dc.subject.otherIndex hypocalcemia
dc.subject.otherIndex influenza
dc.subject.otherIndex iron chelation
dc.subject.otherIndex iron overload
dc.subject.otherIndex kidney failure
dc.subject.otherIndex major clinical study
dc.subject.otherIndex male
dc.subject.otherIndex Middle East
dc.subject.otherIndex nausea
dc.subject.otherIndex obstructive jaundice
dc.subject.otherIndex preschool child
dc.subject.otherIndex priority journal
dc.subject.otherIndex side effect
dc.subject.otherIndex splenomegaly
dc.subject.otherIndex treatment response
dc.subject.otherIndex vomiting
dc.subject.otherIndex Adolescent
dc.subject.otherIndex Adult
dc.subject.otherIndex Benzoic Acids
dc.subject.otherIndex beta-Thalassemia
dc.subject.otherIndex Child, Preschool
dc.subject.otherIndex Deferoxamine
dc.subject.otherIndex Drug Toxicity
dc.subject.otherIndex Female
dc.subject.otherIndex Ferritins
dc.subject.otherIndex Humans
dc.subject.otherIndex Iron
dc.subject.otherIndex Iron Chelating Agents
dc.subject.otherIndex Iron Overload
dc.subject.otherIndex Liver
dc.subject.otherIndex Male
dc.subject.otherIndex Pyridones
dc.subject.otherIndex Treatment Outcome
dc.subject.otherIndex Triazoles
dc.subject.otherIndex Young Adult
dc.subject.otherKeywordPlus CHRONIC HEPATITIS
dc.subject.otherKeywordPlus THERAPY
dc.subject.otherKeywordPlus COMPLICATIONS
dc.subject.otherKeywordPlus DEFEROXAMINE
dc.subject.otherKeywordPlus SURVIVAL
dc.subject.otherKeywordPlus ICL670
dc.subject.otherWOS Hematology


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