Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS study

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dc.contributor.authorTaher, Ali T.
dc.contributor.authorCappellini, M. Domenica
dc.contributor.authorAydinok, Yesim
dc.contributor.authorPorter, John B.
dc.contributor.authorKarakas, Zeynep
dc.contributor.authorViprakasit, Vip
dc.contributor.authorSiritanaratkul, Noppadol
dc.contributor.authorKattamis, Antonis
dc.contributor.authorWang, Candace
dc.contributor.authorZhu, Zewen
dc.contributor.authorJoaquin, Victor
dc.contributor.authorUwamahoro, Marie Jose
dc.contributor.authorLai, Yong-Rong
dc.date.accessioned2019-10-27T23:11:13Z
dc.date.available2019-10-27T23:11:13Z
dc.date.issued2016
dc.departmentEge Üniversitesien_US
dc.description.abstractEfficacy and safety of iron chelation therapy with deferasirox in iron-overloaded non-transfusion-dependent thalassaemia (NTDT) patients were established in the THALASSA study. THETIS, an open-label, single-arm, multicentre, Phase IV study, added to this evidence by investigating earlier dose escalation by baseline liver iron concentration (LIC) (week 4: escalation according to baseline LIC; week 24: adjustment according to LIC response, maximum 30 mg/kg/day). The primary efficacy endpoint was absolute change in LIC from baseline to week 52. 134 iron-overloaded non-transfusion-dependent anaemia patients were enrolled and received deferasirox starting at 10 mg/kg/day. Mean actual dose +/- SD over 1 year was 14.70 +/- 5.48 mg/kg/day. At week 52, mean LIC SD decreased significantly from 15.13 +/- 10.72 mg Fe/g dw at baseline to 8.46 +/- 6.25 mg Fe/g dw (absolute change from baseline, -6.68 +/- 7.02 mg Fe/g dw [95% CI: -7.91, -5.45]; P<0.0001). Most common drug-related adverse events were gastrointestinal: abdominal discomfort, diarrhoea and nausea (n = 6 each). There was one death (pneumonia, not considered drug related). With significant and clinically relevant reductions in iron burden alongside a safety profile similar to that in THALASSA, these data support earlier escalation with higher deferasirox doses in iron-overloaded non-transfusion-dependent anaemia patients. (C) 2015 The Authors. Published by Elsevier Ltd.en_US
dc.description.sponsorshipNovartis Pharma AG; Novartis Pharmaceuticalsen_US
dc.description.sponsorshipThis study was funded by Novartis Pharma AG. We thank Rebecca Helson, PhD, of Mudskipper Business Ltd. for medical editorial assistance. Financial support for medical editorial assistance was provided by Novartis Pharmaceuticals.en_US
dc.identifier.doi10.1016/j.bcmd.2015.11.002en_US
dc.identifier.endpage29en_US
dc.identifier.issn1079-9796
dc.identifier.issn1096-0961
dc.identifier.pmid26852651en_US
dc.identifier.scopusqualityQ2en_US
dc.identifier.startpage23en_US
dc.identifier.urihttps://doi.org/10.1016/j.bcmd.2015.11.002
dc.identifier.urihttps://hdl.handle.net/11454/53038
dc.identifier.volume57en_US
dc.identifier.wosWOS:000370467700004en_US
dc.identifier.wosqualityQ3en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherAcademic Press Inc Elsevier Scienceen_US
dc.relation.ispartofBlood Cells Molecules and Diseasesen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.subjectNon-transfusion-dependent thalassaemiaen_US
dc.subjectNTDTen_US
dc.subjectIron overloaden_US
dc.subjectIron chelation therapyen_US
dc.subjectDose escalationen_US
dc.titleOptimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS studyen_US
dc.typeArticleen_US

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