Observational study comparing long-term safety and efficacy of Deferasirox with Desferrioxamine therapy in chelation-naïve children with transfusional iron overload

European Journal of Haematology
Yesim AydinokAkif Yesilipek

Abstract

An observational study was conducted to explore postmarketing safety and efficacy of Deferasirox (DFX) in comparison with conventional Desferrioxamine (DFO) in chelation-naïve children with transfusional iron overload. Transfusion-dependent children (aged ≤ 5 yr) who had serum ferritin above 1000 μg/L and had been prescribed either first-line DFX or DFO for at least 12 months to maintain serum ferritin between 500 and 1000 μg/L were included. Initial DFX dose was 20 mg/kg/d for 7 d a week, and DFO dose was 25-35 mg/kg/d subcutaneously, given for 5 d a week. Dose adjustments were based on serum ferritin changes and safety markers. The primary efficacy endpoint was change in serum ferritin from baseline. The effect of transfusional iron loading rate (ILR) and different doses of chelators on serum ferritin was also assessed. A total of 111 patients were observed for a median of 2.29 yr on DFX (n = 71) and 2.75 yr on DFO (n = 40). Absolute change in serum ferritin from baseline to the last available observation was not significant with DFX (91 μg/L, P = 0.5) but significantly higher with DFO (385 μg/L, P < 0.005). ILR and DFX doses had a major impact on serum ferritin changes in DFX cohort. The height- and weight-standard deviation...Continue Reading

References

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Citations

Jun 6, 2013·PloS One·Ji Won LeeHyo Seop Ahn
Jul 23, 2014·Nature Reviews. Nephrology·Juan Daniel Díaz-GarcíaAlberto Ortiz
Jun 13, 2012·European Journal of Haematology·Sunil Bhandari, Renzo Galanello
Mar 22, 2014·British Journal of Haematology·Yesim AydinokVip Viprakasit
Apr 1, 2015·Future Medicinal Chemistry·M Amélia Santos, Sílvia Chaves
Mar 8, 2016·American Journal of Hematology·Raffaella OrigaCarlo Dessì
Nov 1, 2016·Pediatric Allergy and Immunology : Official Publication of the European Society of Pediatric Allergy and Immunology·Grace Isobel DaviesDonald Bowden
Nov 29, 2016·European Journal of Haematology·Sebastian BotzenhardtAntje Neubert
Oct 21, 2016·The Journal of Pharmacy and Pharmacology·Sarah AllegraAntonio D'Avolio
Dec 6, 2017·Clinical and Experimental Pharmacology & Physiology·Sarah AllegraAntonio Piga

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