Publication: Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS study
Issued Date
2016-03-01
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ISSN
10960961
10799796
10799796
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2-s2.0-84959208200
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Mahidol University
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SCOPUS
Bibliographic Citation
Blood Cells, Molecules, and Diseases. Vol.57, (2016), 23-29
Suggested Citation
Ali T. Taher, M. Domenica Cappellini, Yesim Aydinok, John B. Porter, Zeynep Karakas, Vip Viprakasit, Noppadol Siritanaratkul, Antonis Kattamis, Candace Wang, Zewen Zhu, Victor Joaquin, Marie José Uwamahoro, Yong Rong Lai Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS study. Blood Cells, Molecules, and Diseases. Vol.57, (2016), 23-29. doi:10.1016/j.bcmd.2015.11.002 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/43084
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Title
Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS study
Abstract
© 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.