Ali T. TaherJohn PorterVip ViprakasitAntonis KattamisSuporn ChuncharuneePranee SutcharitchanNoppadol SiritanaratkulRenzo GalanelloZeynep KarakasTomasz LawniczekJacqueline RosYiyun ZhangDany HabrMaria Domenica CappelliniAmerican University of BeirutUCLMahidol UniversityUniversity of AthensChulalongkorn UniversityUniversita degli Studi di CagliariIstanbul Tip FakultesiNovartis International AGNovartis PharmaceuticalsUniversita degli Studi di Milano2018-06-112018-06-112012-08-02Blood. Vol.120, No.5 (2012), 970-97715280020000649712-s2.0-84864530218https://repository.li.mahidol.ac.th/handle/20.500.14594/13643Nontransfusion-dependent thalassemia (NTDT) patients may develop iron overload and its associated complications despite receiving only occasional or no transfusions. The present 1-year, randomized, double-blind, placebo-controlled THALASSA (Assessment of Exjade in Nontransfusion-Dependent Thalassemia) trial assessed the efficacy and safety of deferasirox in iron-overloaded NTDT patients. Atotal of 166 patients were randomized in a 2:1:2:1 ratio to starting doses of 5 or 10 mg/kg/d of deferasirox or placebo. The means ± SD of the actual deferasirox doses received over the duration of the study in the 5 and 10 mg/kg/d starting dose cohorts were 5.7 ± 1.4 and 11.5 ± 2.9 mg/kg/d, respectively. At 1 year, the liver iron concentration (LIC) decreased significantly compared with placebo (least-squares mean [LSM] ± SEM, -2.33 ± 0.7 mg Fe/g dry weight [dw] , P = .001, and -4.18 ± 0.69 mg Fe/g dw, P < .001) for the 5 and 10 mg/kg/d deferasirox groups, respectively (baseline values [means ± SD], 13.11 ± 7.29 and 14.56 ± 7.92 mg Fe/g dw, respectively). Similarly, serum ferritin decreased significantly compared with placebo by LSM -235 and -337 ng/mL for the deferasirox 5 and 10 mg/kg/d groups, respectively (P < .001). In the placebo patients, LIC and serum ferritin increased from baseline by 0.38 mg Fe/g dw and 115 ng/mL (LSM), respectively. The most common drug-related adverse events were nausea (n = 11; 6.6%), rash (n = 8; 4.8%), and diarrhea (n = 6; 3.6%). This is the first randomized study showing that iron chelation with deferasirox significantly reduces iron overload in NTDT patients with a frequency of overall adverse events similar to placebo. © 2012 by The American Society of Hematology.Mahidol UniversityBiochemistry, Genetics and Molecular BiologyImmunology and MicrobiologyMedicineArticleSCOPUS10.1182/blood-2012-02-412692