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dc.contributor.authorAli T. Taheren_US
dc.contributor.authorJohn Porteren_US
dc.contributor.authorVip Viprakasiten_US
dc.contributor.authorAntonis Kattamisen_US
dc.contributor.authorSuporn Chuncharuneeen_US
dc.contributor.authorPranee Sutcharitchanen_US
dc.contributor.authorNoppadol Siritanaratkulen_US
dc.contributor.authorRenzo Galanelloen_US
dc.contributor.authorZeynep Karakasen_US
dc.contributor.authorTomasz Lawniczeken_US
dc.contributor.authorJacqueline Rosen_US
dc.contributor.authorYiyun Zhangen_US
dc.contributor.authorDany Habren_US
dc.contributor.authorMaria Domenica Cappellinien_US
dc.contributor.otherAmerican University of Beiruten_US
dc.contributor.otherUCLen_US
dc.contributor.otherMahidol Universityen_US
dc.contributor.otherUniversity of Athensen_US
dc.contributor.otherChulalongkorn Universityen_US
dc.contributor.otherUniversita degli Studi di Cagliarien_US
dc.contributor.otherIstanbul Tip Fakultesien_US
dc.contributor.otherNovartis International AGen_US
dc.contributor.otherNovartis Pharmaceuticalsen_US
dc.contributor.otherUniversita degli Studi di Milanoen_US
dc.date.accessioned2018-06-11T04:34:41Z-
dc.date.available2018-06-11T04:34:41Z-
dc.date.issued2012-08-02en_US
dc.identifier.citationBlood. Vol.120, No.5 (2012), 970-977en_US
dc.identifier.issn15280020en_US
dc.identifier.issn00064971en_US
dc.identifier.other2-s2.0-84864530218en_US
dc.identifier.urihttps://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=84864530218&origin=inwarden_US
dc.identifier.urihttp://repository.li.mahidol.ac.th/dspace/handle/123456789/13643-
dc.description.abstractNontransfusion-dependent thalassemia (NTDT) patients may develop iron overload and its associated complications despite receiving only occasional or no transfusions. The present 1-year, randomized, double-blind, placebo-controlled THALASSA (Assessment of Exjade in Nontransfusion-Dependent Thalassemia) trial assessed the efficacy and safety of deferasirox in iron-overloaded NTDT patients. Atotal of 166 patients were randomized in a 2:1:2:1 ratio to starting doses of 5 or 10 mg/kg/d of deferasirox or placebo. The means ± SD of the actual deferasirox doses received over the duration of the study in the 5 and 10 mg/kg/d starting dose cohorts were 5.7 ± 1.4 and 11.5 ± 2.9 mg/kg/d, respectively. At 1 year, the liver iron concentration (LIC) decreased significantly compared with placebo (least-squares mean [LSM] ± SEM, -2.33 ± 0.7 mg Fe/g dry weight [dw] , P = .001, and -4.18 ± 0.69 mg Fe/g dw, P < .001) for the 5 and 10 mg/kg/d deferasirox groups, respectively (baseline values [means ± SD], 13.11 ± 7.29 and 14.56 ± 7.92 mg Fe/g dw, respectively). Similarly, serum ferritin decreased significantly compared with placebo by LSM -235 and -337 ng/mL for the deferasirox 5 and 10 mg/kg/d groups, respectively (P < .001). In the placebo patients, LIC and serum ferritin increased from baseline by 0.38 mg Fe/g dw and 115 ng/mL (LSM), respectively. The most common drug-related adverse events were nausea (n = 11; 6.6%), rash (n = 8; 4.8%), and diarrhea (n = 6; 3.6%). This is the first randomized study showing that iron chelation with deferasirox significantly reduces iron overload in NTDT patients with a frequency of overall adverse events similar to placebo. © 2012 by The American Society of Hematology.en_US
dc.rightsMahidol Universityen_US
dc.source.urihttps://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=84864530218&origin=inwarden_US
dc.subjectBiochemistry, Genetics and Molecular Biologyen_US
dc.subjectImmunology and Microbiologyen_US
dc.subjectMedicineen_US
dc.titleDeferasirox reduces iron overload significantly in nontransfusion-dependent thalassemia: 1-Year results from a prospective, randomized, double-blind, placebo-controlled studyen_US
dc.typeArticleen_US
dc.rights.holderSCOPUSen_US
dc.identifier.doi10.1182/blood-2012-02-412692en_US
Appears in Collections:Scopus 2011-2015

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