Ali T. TaherJohn B. PorterVip ViprakasitAntonis KattamisSuporn ChuncharuneePranee SutcharitchanNoppadol SiritanaratkulRenzo GalanelloZeynep KarakasTomasz LawniczekDany HabrJacqueline RosYiyun ZhangM. Domenica CappelliniAmerican University of Beirut Medical CenterUCL Cancer InstituteMahidol UniversityUniversity of AthensChulalongkorn UniversityOspedale Regional MicrocitemieIstanbul Tip FakultesiNovartis International AGNovartis PharmaceuticalsUniversita degli Studi di Milano2018-10-192018-10-192013-06-01American Journal of Hematology. Vol.88, No.6 (2013), 503-50610968652036186092-s2.0-84878169574https://repository.li.mahidol.ac.th/handle/20.500.14594/32328The 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.Mahidol UniversityMedicineDeferasirox demonstrates a dose-dependent reduction in liver iron concentration and consistent efficacy across subgroups of non-transfusion-dependent thalassemia patientsArticleSCOPUS10.1002/ajh.23445