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

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

Research output: Contribution to journalArticle

13 Citations (Scopus)

Abstract

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.

Original languageEnglish
Pages (from-to)23-29
Number of pages7
JournalBlood cells, molecules & diseases
Volume57
DOIs
Publication statusPublished - Mar 1 2016

Fingerprint

Chelation Therapy
Thalassemia
Iron
Liver
Anemia
deferasirox
Safety
Drug-Related Side Effects and Adverse Reactions
Nausea
Diarrhea
Pneumonia

Keywords

  • Dose escalation
  • Iron chelation therapy
  • Iron overload
  • Non-transfusion-dependent thalassaemia
  • NTDT

ASJC Scopus subject areas

  • Molecular Biology
  • Molecular Medicine
  • Hematology
  • Cell Biology

Cite this

Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients : 1-year results from the THETIS study. / Taher, Ali T.; Cappellini, M. Domenica; Aydinok, Yesim; Porter, John B.; Karakas, Zeynep; Viprakasit, Vip; Siritanaratkul, Noppadol; Kattamis, Antonis; Wang, Candace; Zhu, Zewen; Joaquin, Victor; Uwamahoro, Marie José; Lai, Yong Rong.

In: Blood cells, molecules & diseases, Vol. 57, 01.03.2016, p. 23-29.

Research output: Contribution to journalArticle

Taher, AT, Cappellini, MD, Aydinok, Y, Porter, JB, Karakas, Z, Viprakasit, V, Siritanaratkul, N, Kattamis, A, Wang, C, Zhu, Z, Joaquin, V, Uwamahoro, MJ & Lai, YR 2016, 'Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS study', Blood cells, molecules & diseases, vol. 57, pp. 23-29. https://doi.org/10.1016/j.bcmd.2015.11.002
Taher, Ali T. ; Cappellini, M. Domenica ; Aydinok, Yesim ; Porter, John B. ; Karakas, Zeynep ; Viprakasit, Vip ; Siritanaratkul, Noppadol ; Kattamis, Antonis ; Wang, Candace ; Zhu, Zewen ; Joaquin, Victor ; Uwamahoro, Marie José ; Lai, Yong Rong. / Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients : 1-year results from the THETIS study. In: Blood cells, molecules & diseases. 2016 ; Vol. 57. pp. 23-29.
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