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Efficacy of Deferasirox for the treatment of iron overload in Chinese thalassaemia major patients: results from a prospective, open‐label, multicentre clinical trial

Lai, Y. ‐R. ; Liu, R. ‐R. ; Li, C. ‐F. ; Huang, S. ‐L. ; Li, Q. ; Habr, D. ; Martin, N. ; Shen, Z. ‐X.

Transfusion Medicine, December 2013, Vol.23(6), pp.389-396 [Periódico revisado por pares]

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  • Título:
    Efficacy of Deferasirox for the treatment of iron overload in Chinese thalassaemia major patients: results from a prospective, open‐label, multicentre clinical trial
  • Autor: Lai, Y. ‐R. ; Liu, R. ‐R. ; Li, C. ‐F. ; Huang, S. ‐L. ; Li, Q. ; Habr, D. ; Martin, N. ; Shen, Z. ‐X.
  • Assuntos: Deferasirox ; Iron Chelation ; Iron Overload ; Serum Ferritin ; Thalassaemia Major
  • É parte de: Transfusion Medicine, December 2013, Vol.23(6), pp.389-396
  • Descrição: Byline: Y.-R. Lai, R.-R. Liu, C.-F. Li, S.-L. Huang, Q. Li, D. Habr, N. Martin, Z.-X. Shen Keywords: deferasirox; iron chelation; iron overload; serum ferritin; thalassaemia major SUMMARY Objective To assess the efficacy and safety of deferasirox in Chinese thalassaemia major (TM) patients Background EPIC (Evaluation of Patients' Iron Chelation with Exjade.sub.[R]) was a large multi-national study and, notably, the first clinical trial of an iron chelator registered with the Chinese State Food and Drug Administration. Methods Efficacy and safety of deferasirox were compared in Chinese (n=117) and non-Chinese (n=998) TM patients. Deferasirox was initiated at 20mgkg.sub.-1day.sub.-1, with titration increments of 5-10 mgkg.sub.-1day.sub.-1, based on serum ferritin trends and safety parameters. Results At baseline, Chinese patients were younger than non-Chinese (mean age 6ae8 versus 19ae5years), with higher median serum ferritin (4519 vs 3058ngmL.sub.-1). Over 1year, mean actual deferasirox dose was similar for Chinese and non-Chinese patients (24ae6 and 24ae0mgkg.sub.-1day.sub.-1, respectively); median serum ferritin did not change significantly from baseline in Chinese patients (+340ngmL.sub.-1, P=0ae102) and significantly decreased in non-Chinese patients (-220ngmL.sub.-1; P 0ae001). In the 1-year extension in Chinese patients, (mean actual deferasirox dose 33ae6mgkg.sub.-1day.sub.-1), median serum ferritin decreased (-756ngmL.sub.-1; P = 0ae0397), with a numerically higher reduction in patients aged a[yen]6 to 12 than 6years (-982 vs -457ngmL.sub.-1, respectively). The safety profile of deferasirox in Chinese patients was similar to the overall population with respect to clinically-relevant findings. Conclusion Age and deferasirox exposure influenced study findings, supporting the need for longer-term treatment and dose escalation to a[yen]30mgkg.sub.-1day.sub.-1 to achieve neutral or negative iron balance in heavily iron overloaded and younger Chinese patients.

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