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Title: Deferasirox demonstrates a dose-dependent reduction in liver iron concentration and consistent efficacy across subgroups of non-transfusion-dependent thalassemia patients
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
Yiyun Zhang
M. Domenica Cappellini
American University of Beirut Medical Center
UCL Cancer Institute
Mahidol University
University of Athens
Chulalongkorn University
Ospedale Regional Microcitemie
Istanbul Tip Fakultesi
Novartis International AG
Novartis Pharmaceuticals
Universita degli Studi di Milano
Keywords: Medicine
Issue Date: 1-Jun-2013
Citation: American Journal of Hematology. Vol.88, No.6 (2013), 503-506
Abstract: The 1-year THALASSA study enrolled 166 patients with various non-transfusion-dependent thalassemia (NTDT) syndromes, degrees of iron burden and patient characteristics, and demonstrated the overall efficacy and safety of deferasirox in reducing liver iron concentration (LIC) in these patients. Here, reduction in LIC with deferasirox 5 and 10mg/kg/day starting dose groups is shown to be consistent across the following patient subgroups-baseline LIC/serum ferritin, age, gender, race, splenectomy (yes/no), and underlying NTDT syndrome (β-thalassemia intermedia, HbE/β-thalassemia or α-thalassemia). These analyses also evaluated deferasirox dosing strategies for patients with NTDT. Greater reductions in LIC were achieved in patients dose-escalated at Week 24 from deferasirox 10mg/kg/day starting dose to 20mg/kg/day. Patients who received an average actual dose of deferasirox >12.5-≤17.5mg/kg/day achieved a greater LIC decrease compared with the ≥7.5-≤12.5mg/kg/day and >0-<7.5mg/kg/day subgroups, demonstrating a dose-response efficacy. LIC reduction across patient subgroups was generally consistent with the primary efficacy analysis with a similar safety profile. © 2013 Wiley Periodicals, Inc.
ISSN: 10968652
Appears in Collections:Scopus 2011-2015

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