Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia

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dc.contributor.authorPennell, Dudley J.
dc.contributor.authorPorter, John B.
dc.contributor.authorCappellini, Maria Domenica
dc.contributor.authorEl-Beshlawy, Amal
dc.contributor.authorChan, Lee Lee
dc.contributor.authorAydinok, Yesim
dc.contributor.authorElalfy, Mohsen Saleh
dc.contributor.authorSutcharitchan, Pranee
dc.contributor.authorLi, Chi-Kong
dc.contributor.authorIbrahim, Hishamshah
dc.contributor.authorViprakasit, Vip
dc.contributor.authorKattamis, Antonis
dc.contributor.authorSmith, Gillian
dc.contributor.authorHabr, Dany
dc.contributor.authorDomokos, Gabor
dc.contributor.authorRoubert, Bernard
dc.contributor.authorTaher, Ali
dc.date.accessioned2019-10-27T21:18:35Z
dc.date.available2019-10-27T21:18:35Z
dc.date.issued2010
dc.departmentEge Üniversitesien_US
dc.description.abstractCardiac iron overload causes most deaths in beta-thalassemia major. The efficacy of deferasirox in reducing or preventing cardiac iron overload was assessed in 192 patients with beta-thalassemia in a 1-year prospective, multicenter study. The cardiac iron reduction arm (n = 114) included patients with magnetic resonance myocardial T2* from 5 to 20 ms (indicating cardiac siderosis), left ventricular ejection fraction (LVEF) of 56% or more, serum ferritin more than 2500 ng/mL, liver iron concentration more than 10 mg Fe/g dry weight, and more than 50 transfused blood units. The prevention arm (n = 78) included otherwise eligible patients whose myocardial T2* was 20 ms or more. The primary end point was the change in myocardial T2* at 1 year. In the cardiac iron reduction arm, the mean deferasirox dose was 32.6 mg/kg per day. Myocardial T2* (geometric mean +/- coefficient of variation) improved from a baseline of 11.2 ms (+/- 40.5%) to 12.9 ms (+/- 49.5%) (+ 16%; P < .001). LVEF (mean +/- SD) was unchanged: 67.4 (+/- 5.7%) to 67.0 (+/- 6.0%) (-0.3%; P = .53). In the prevention arm, baseline myocardial T2* was unchanged from baseline of 32.0 ms (+/- 25.6%) to 32.5 ms (+/- 25.1%) (+ 2%; P = .57) and LVEF increased from baseline 67.7 (+/- 4.7%) to 69.6 (+/- 4.5%) (+ 1.8%; P < .001). This prospective study shows that deferasirox is effective in removing and preventing myocardial iron accumulation. This study is registered at http://clinicaltrials.gov as NCT00171821. (Blood. 2010; 115: 2364-2371)en_US
dc.description.sponsorshipNovartis Pharma AG; Novartis Pharmaceuticals; British Heart FoundationBritish Heart Foundation [PG/09/074/27961]; National Institute for Health ResearchNational Institute for Health Research (NIHR) [NF-SI-0508-10081]en_US
dc.description.sponsorshipThis study was sponsored by Novartis Pharma AG. Financial support for medical editorial assistance was provided by Novartis Pharmaceuticals.en_US
dc.identifier.doi10.1182/blood-2009-04-217455en_US
dc.identifier.endpage2371en_US
dc.identifier.issn0006-4971
dc.identifier.issn1528-0020
dc.identifier.issue12en_US
dc.identifier.pmid19996412en_US
dc.identifier.startpage2364en_US
dc.identifier.urihttps://doi.org/10.1182/blood-2009-04-217455
dc.identifier.urihttps://hdl.handle.net/11454/44007
dc.identifier.volume115en_US
dc.identifier.wosWOS:000275981900009en_US
dc.identifier.wosqualityQ1en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherAmer Soc Hematologyen_US
dc.relation.ispartofBlooden_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.titleEfficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemiaen_US
dc.typeArticleen_US

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