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Deferasirox demonstrates a dose‐dependent reduction in liver iron concentration and consistent efficacy across subgroups of non‐transfusion‐dependent thalassemia patients
Author(s) -
Taher Ali T.,
Porter John B.,
Viprakasit Vip,
Kattamis Antonis,
Chuncharunee Suporn,
Sutcharitchan Pranee,
Siritanaratkul Noppadol,
Galanello Renzo,
Karakas Zeynep,
Lawniczek Tomasz,
Habr Dany,
Ros Jacqueline,
Zhang Yiyun,
Cappellini M. Domenica
Publication year - 2013
Publication title -
american journal of hematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.456
H-Index - 105
eISSN - 1096-8652
pISSN - 0361-8609
DOI - 10.1002/ajh.23445
Subject(s) - deferasirox , medicine , thalassemia , dosing , gastroenterology , beta thalassemia , deferoxamine , adverse effect , splenectomy , pediatrics , spleen
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 10 mg/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 10 mg/kg/day starting dose to 20 mg/kg/day. Patients who received an average actual dose of deferasirox >12.5–≤17.5 mg/kg/day achieved a greater LIC decrease compared with the ≥7.5–≤12.5 mg/kg/day and >0–<7.5 mg/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. Am. J. Hematol. 88:503–506, 2013. © 2013 Wiley Periodicals, Inc.