Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS study

Taher A. T., Cappellini M. D., AYDINOK Y., Porter J. B., Karakas Z., Viprakasit V., ...Daha Fazla

BLOOD CELLS MOLECULES AND DISEASES, cilt.57, ss.23-29, 2016 (SCI-Expanded) identifier identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 57
  • Basım Tarihi: 2016
  • Doi Numarası: 10.1016/j.bcmd.2015.11.002
  • Dergi Adı: BLOOD CELLS MOLECULES AND DISEASES
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Sayfa Sayıları: ss.23-29
  • Anahtar Kelimeler: Non-transfusion-dependent thalassaemia, NTDT, Iron overload, Iron chelation therapy, Dose escalation, SERUM FERRITIN, OVERLOAD, DEFERIPRONE, MORBIDITY
  • İstanbul Üniversitesi Adresli: Evet

Özet

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. (C) 2015 The Authors. Published by Elsevier Ltd.