Deferasirox demonstrates a dose-dependent reduction in liver iron concentration and consistent efficacy across subgroups of non-transfusion-dependent thalassemia patients.

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 subgroupsbaseline LIC/serum ferritin, age, gender, race, splenectomy (yes/no), and underlying NTDT syndrome (-thalassemia intermedia, HbE/-thalassemia or -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 >12.517.5mg/kg/day achieved a greater LIC decrease compared with the 7.512.5mg/kg/day and >0<7.5mg/kg/day subgroups, demonstrating a doseresponse efficacy. LIC reduction across patient subgroups was generally consistent with the primary efficacy analysis with a similar safety profile. Am. J. Hematol. 88:503506, 2013. (c) 2013 Wiley Periodicals, Inc.