Pharmacological and clinical evaluation of deferasirox formulations for treatment tailoring

Abstract Deferasirox (DFX) is the newest among three different chelators available to treat iron overload in iron-loading anaemias, firstly released as Dispersible Tablets (DT) and more recently replaced by Film-Coated Tablets (FCT). In this retrospective observational study, pharmacokinetics, pharm...

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Autores principales:	Andrea Piolatto, Paola Berchialla, Sarah Allegra, Silvia De Francia, Giovanni Battista Ferrero, Antonio Piga, Filomena Longo
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/5ec962bf5733487bb7ac0972af87b973
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spelling	oai:doaj.org-article:5ec962bf5733487bb7ac0972af87b9732021-12-02T17:41:04ZPharmacological and clinical evaluation of deferasirox formulations for treatment tailoring10.1038/s41598-021-91983-w2045-2322https://doaj.org/article/5ec962bf5733487bb7ac0972af87b9732021-06-01T00:00:00Zhttps://doi.org/10.1038/s41598-021-91983-whttps://doaj.org/toc/2045-2322Abstract Deferasirox (DFX) is the newest among three different chelators available to treat iron overload in iron-loading anaemias, firstly released as Dispersible Tablets (DT) and more recently replaced by Film-Coated Tablets (FCT). In this retrospective observational study, pharmacokinetics, pharmacodynamics, and safety features of DFX treatment were analyzed in 74 patients that took both formulations subsequently under clinical practice conditions. Bioavailability of DFX FCT compared to DT resulted higher than expected [Cmax: 99.5 (FCT) and 69.7 (DT) μMol/L; AUC: 1278 (FCT) and 846 (DT), P < 0.0001]. DFX FCT was also superior in scalability among doses. After one year of treatment for each formulation, no differences were observed between the treatments in the overall iron overload levels; however, DFX FCT but not DT showed a significant dose–response correlation [Spearman r (dose-serum ferritin variation): − 0.54, P < 0.0001]. Despite being administered at different dosages, the long-term safety profile was not different between formulations: a significant increase in renal impairment risk was observed for both treatments and it was reversible under strict monitoring (P < 0.002). Altogether, these data constitute a comprehensive comparison of DFX formulations in thalassaemia and other iron-loading anaemias, confirming the effectiveness and safety characteristics of DFX and its applicability for treatment tailoring.Andrea PiolattoPaola BerchiallaSarah AllegraSilvia De FranciaGiovanni Battista FerreroAntonio PigaFilomena LongoNature PortfolioarticleMedicineRScienceQENScientific Reports, Vol 11, Iss 1, Pp 1-12 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Andrea Piolatto Paola Berchialla Sarah Allegra Silvia De Francia Giovanni Battista Ferrero Antonio Piga Filomena Longo Pharmacological and clinical evaluation of deferasirox formulations for treatment tailoring
description	Abstract Deferasirox (DFX) is the newest among three different chelators available to treat iron overload in iron-loading anaemias, firstly released as Dispersible Tablets (DT) and more recently replaced by Film-Coated Tablets (FCT). In this retrospective observational study, pharmacokinetics, pharmacodynamics, and safety features of DFX treatment were analyzed in 74 patients that took both formulations subsequently under clinical practice conditions. Bioavailability of DFX FCT compared to DT resulted higher than expected [Cmax: 99.5 (FCT) and 69.7 (DT) μMol/L; AUC: 1278 (FCT) and 846 (DT), P < 0.0001]. DFX FCT was also superior in scalability among doses. After one year of treatment for each formulation, no differences were observed between the treatments in the overall iron overload levels; however, DFX FCT but not DT showed a significant dose–response correlation [Spearman r (dose-serum ferritin variation): − 0.54, P < 0.0001]. Despite being administered at different dosages, the long-term safety profile was not different between formulations: a significant increase in renal impairment risk was observed for both treatments and it was reversible under strict monitoring (P < 0.002). Altogether, these data constitute a comprehensive comparison of DFX formulations in thalassaemia and other iron-loading anaemias, confirming the effectiveness and safety characteristics of DFX and its applicability for treatment tailoring.
format	article
author	Andrea Piolatto Paola Berchialla Sarah Allegra Silvia De Francia Giovanni Battista Ferrero Antonio Piga Filomena Longo
author_facet	Andrea Piolatto Paola Berchialla Sarah Allegra Silvia De Francia Giovanni Battista Ferrero Antonio Piga Filomena Longo
author_sort	Andrea Piolatto
title	Pharmacological and clinical evaluation of deferasirox formulations for treatment tailoring
title_short	Pharmacological and clinical evaluation of deferasirox formulations for treatment tailoring
title_full	Pharmacological and clinical evaluation of deferasirox formulations for treatment tailoring
title_fullStr	Pharmacological and clinical evaluation of deferasirox formulations for treatment tailoring
title_full_unstemmed	Pharmacological and clinical evaluation of deferasirox formulations for treatment tailoring
title_sort	pharmacological and clinical evaluation of deferasirox formulations for treatment tailoring
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/5ec962bf5733487bb7ac0972af87b973
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Pharmacological and clinical evaluation of deferasirox formulations for treatment tailoring

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