Ali T. TaherM. Domenica CappelliniYesim AydinokJohn B. PorterZeynep KarakasVip ViprakasitNoppadol SiritanaratkulAntonis KattamisCandace WangZewen ZhuVictor JoaquinMarie José UwamahoroYong Rong LaiAmerican University of Beirut Medical CenterUniversità degli Studi di MilanoEge University Medical SchoolUCLIstanbul Tip FakultesiMahidol UniversityUniversity of AthensNovartis PharmaceuticalsNovartis International AGGuangxi Medical University2018-12-112019-03-142018-12-112019-03-142016-03-01Blood Cells, Molecules, and Diseases. Vol.57, (2016), 23-2910960961107997962-s2.0-84959208200https://repository.li.mahidol.ac.th/handle/20.500.14594/43084© 2015 The Authors. Efficacy and safety of iron chelation therapy with deferasirox in iron-overloaded non-transfusion-dependent thalassaemia (NTDT) patients were established in the THALASSA study. THETIS, an open-label, single-arm, multicentre, Phase IV study, added to this evidence by investigating earlier dose escalation by baseline liver iron concentration (LIC) (week 4: escalation according to baseline LIC; week 24: adjustment according to LIC response, maximum 30 mg/kg/day). The primary efficacy endpoint was absolute change in LIC from baseline to week 52. 134 iron-overloaded non-transfusion-dependent anaemia patients were enrolled and received deferasirox starting at 10 mg/kg/day. Mean actual dose ± SD over 1 year was 14.70 ± 5.48 mg/kg/day. At week 52, mean LIC ± SD decreased significantly from 15.13 ± 10.72 mg Fe/g dw at baseline to 8.46 ± 6.25 mg Fe/g dw (absolute change from baseline, -6.68 ± 7.02 mg Fe/g dw [95% CI: -7.91, -5.45]; P < 0.0001). Most common drug-related adverse events were gastrointestinal: abdominal discomfort, diarrhoea and nausea (n = 6 each). There was one death (pneumonia, not considered drug related). With significant and clinically relevant reductions in iron burden alongside a safety profile similar to that in THALASSA, these data support earlier escalation with higher deferasirox doses in iron-overloaded non-transfusion-dependent anaemia patients.Mahidol UniversityBiochemistry, Genetics and Molecular BiologyArticleSCOPUS10.1016/j.bcmd.2015.11.002