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Please use this identifier to cite or link to this item: http://repository.li.mahidol.ac.th/dspace/handle/123456789/32100
Title: Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients: 1-year extension results from the THALASSA study
Authors: Ali T. Taher
John B. Porter
Vip Viprakasit
Antonis Kattamis
Suporn Chuncharunee
Pranee Sutcharitchan
Noppadol Siritanaratkul
Renzo Galanello
Zeynep Karakas
Tomasz Lawniczek
Dany Habr
Jacqueline Ros
Zewen Zhu
M. Domenica Cappellini
American University of Beirut
UCL
Mahidol University
University of Athens
Chulalongkorn University
Universita degli Studi di Cagliari
Istanbul Tip Fakultesi
Novartis International AG
Novartis Pharmaceuticals
Universita degli Studi di Milano
Keywords: Medicine
Issue Date: 1-Nov-2013
Citation: Annals of Hematology. Vol.92, No.11 (2013), 1485-1493
Abstract: Patients with non-transfusion-dependent thalassemia (NTDT) often develop iron overload that requires chelation to levels below the threshold associated with complications. This can take several years in patients with high iron burden, highlighting the value of long-term chelation data. Here, we report the 1-year extension of the THALASSA trial assessing deferasirox in NTDT; patients continued with deferasirox or crossed from placebo to deferasirox. Of 133 patients entering extension, 130 completed. Liver iron concentration (LIC) continued to decrease with deferasirox over 2 years; mean change was -7.14 mg Fe/g dry weight (dw) (mean dose 9.8 ± 3.6 mg/kg/day). In patients originally randomized to placebo, whose LIC had increased by the end of the core study, LIC decreased in the extension with deferasirox with a mean change of -6.66 mg Fe/g dw (baseline to month 24; mean dose in extension 13.7 ± 4.6 mg/kg/day). Of 166 patients enrolled, 64 (38.6 %) and 24 (14.5 %) patients achieved LIC <5 and <3 mg Fe/g dw by the end of the study, respectively. Mean LIC reduction was greatest in patients with the highest pretreatment LIC. Deferasirox progressively decreases iron overload over 2 years in NTDT patients with both low and high LIC. Safety profile of deferasirox over 2 years was consistent with that in the core study. © 2013 The Author(s).
URI: https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=84885662128&origin=inward
http://repository.li.mahidol.ac.th/dspace/handle/123456789/32100
ISSN: 14320584
09395555
Appears in Collections:Scopus 2011-2015

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