Treatment of hemophilia B: focus on recombinant factor IX

Massimo Franchini, Francesco Frattini, Silvia Crestani, Cinzia Sissa, Carlo BonfantiDepartment of Transfusion Medicine and Hematology, Carlo Poma Hospital, Mantua, ItalyAbstract: Hemophilia B is a recessive X-linked bleeding disorder characterized by deficiency of the coagulation factor IX (FIX). In...

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Autores principales:	Franchini M, Frattini F, Crestani S, Sissa C, Bonfanti C
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2013
Materias:	Medicine (General) R5-920
Acceso en línea:	https://doaj.org/article/c1eeeb6d01c54c84a7493cbb6f11530c
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spelling	oai:doaj.org-article:c1eeeb6d01c54c84a7493cbb6f11530c2021-12-02T05:30:21ZTreatment of hemophilia B: focus on recombinant factor IX1177-54751177-5491https://doaj.org/article/c1eeeb6d01c54c84a7493cbb6f11530c2013-02-01T00:00:00Zhttp://www.dovepress.com/treatment-of-hemophilia-b-focus-on-recombinant-factor-ix-a12186https://doaj.org/toc/1177-5475https://doaj.org/toc/1177-5491Massimo Franchini, Francesco Frattini, Silvia Crestani, Cinzia Sissa, Carlo BonfantiDepartment of Transfusion Medicine and Hematology, Carlo Poma Hospital, Mantua, ItalyAbstract: Hemophilia B is a recessive X-linked bleeding disorder characterized by deficiency of the coagulation factor IX (FIX). In hemophilia B patients the severity of the bleeding phenotype is related to the degree of the FIX defect. Hemophilia B treatment has improved greatly in the last 20 years with the introduction first of plasma-derived and then of recombinant FIX concentrates. Replacement therapy may be administered through on-demand or prophylaxis regimens, but the latter treatment modality has been shown to be superior in prevention of hemophilic arthropathy and in improvement of patients&#39; quality of life. The purpose of this narrative review is to summarize the current knowledge on treatment strategies for hemophilia B, focusing on recombinant FIX products either clinically used or in development. There is only one rFIX product that is licensed to treat hemophilia B patients; from the analysis of the literature data presented in this review, the authors conclude that this rFIX product has demonstrated an excellent safety profile and excellent clinical efficacy for halting and preventing bleeds in hemophilia B patients. While prophylaxis has emerged as the best therapeutic strategy for such patients because of its ability to prevent hemophilic arthropathy and to improve patients&#39; quality of life, the pharmacokinetically tailored dosing of rFIX is another key point when planning hemophilia B treatment, as it allows optimization of the factor concentrate usage. Further clinical studies are needed to better assess the safety and efficacy (ie, the incidence of adverse reactions and inhibitor development) of newer rFIX products.Keywords: recombinant FIX products, plasma-derived FIX concentrate, bleeding, blood clotting disorder, on-demand treatment, prophylaxis treatmentFranchini MFrattini FCrestani SSissa CBonfanti CDove Medical PressarticleMedicine (General)R5-920ENBiologics: Targets & Therapy, Vol 2013, Iss default, Pp 33-38 (2013)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine (General) R5-920
spellingShingle	Medicine (General) R5-920 Franchini M Frattini F Crestani S Sissa C Bonfanti C Treatment of hemophilia B: focus on recombinant factor IX
description	Massimo Franchini, Francesco Frattini, Silvia Crestani, Cinzia Sissa, Carlo BonfantiDepartment of Transfusion Medicine and Hematology, Carlo Poma Hospital, Mantua, ItalyAbstract: Hemophilia B is a recessive X-linked bleeding disorder characterized by deficiency of the coagulation factor IX (FIX). In hemophilia B patients the severity of the bleeding phenotype is related to the degree of the FIX defect. Hemophilia B treatment has improved greatly in the last 20 years with the introduction first of plasma-derived and then of recombinant FIX concentrates. Replacement therapy may be administered through on-demand or prophylaxis regimens, but the latter treatment modality has been shown to be superior in prevention of hemophilic arthropathy and in improvement of patients&#39; quality of life. The purpose of this narrative review is to summarize the current knowledge on treatment strategies for hemophilia B, focusing on recombinant FIX products either clinically used or in development. There is only one rFIX product that is licensed to treat hemophilia B patients; from the analysis of the literature data presented in this review, the authors conclude that this rFIX product has demonstrated an excellent safety profile and excellent clinical efficacy for halting and preventing bleeds in hemophilia B patients. While prophylaxis has emerged as the best therapeutic strategy for such patients because of its ability to prevent hemophilic arthropathy and to improve patients&#39; quality of life, the pharmacokinetically tailored dosing of rFIX is another key point when planning hemophilia B treatment, as it allows optimization of the factor concentrate usage. Further clinical studies are needed to better assess the safety and efficacy (ie, the incidence of adverse reactions and inhibitor development) of newer rFIX products.Keywords: recombinant FIX products, plasma-derived FIX concentrate, bleeding, blood clotting disorder, on-demand treatment, prophylaxis treatment
format	article
author	Franchini M Frattini F Crestani S Sissa C Bonfanti C
author_facet	Franchini M Frattini F Crestani S Sissa C Bonfanti C
author_sort	Franchini M
title	Treatment of hemophilia B: focus on recombinant factor IX
title_short	Treatment of hemophilia B: focus on recombinant factor IX
title_full	Treatment of hemophilia B: focus on recombinant factor IX
title_fullStr	Treatment of hemophilia B: focus on recombinant factor IX
title_full_unstemmed	Treatment of hemophilia B: focus on recombinant factor IX
title_sort	treatment of hemophilia b: focus on recombinant factor ix
publisher	Dove Medical Press
publishDate	2013
url	https://doaj.org/article/c1eeeb6d01c54c84a7493cbb6f11530c
work_keys_str_mv	AT franchinim treatmentofhemophiliabfocusonrecombinantfactorix AT frattinif treatmentofhemophiliabfocusonrecombinantfactorix AT crestanis treatmentofhemophiliabfocusonrecombinantfactorix AT sissac treatmentofhemophiliabfocusonrecombinantfactorix AT bonfantic treatmentofhemophiliabfocusonrecombinantfactorix
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Treatment of hemophilia B: focus on recombinant factor IX

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