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International consensus recommendations on the management of people with haemophilia B

Haemophilia B is a rare X-linked genetic deficiency of coagulation factor IX (FIX) that, if untreated, can cause recurrent and disabling bleeding, potentially leading to severe arthropathy and/or life-threatening haemorrhage. Recent decades have brought significant improvements in haemophilia B mana...

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Autores principales: Hart, Daniel P., Matino, Davide, Astermark, Jan, Dolan, Gerard, d’Oiron, Roseline, Hermans, Cédric, Jiménez-Yuste, Victor, Linares, Adriana, Matsushita, Tadashi, McRae, Simon, Ozelo, Margareth C., Platton, Sean, Stafford, Darrel, Sidonio, Robert F., Tiede, Andreas
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8980430/
https://www.ncbi.nlm.nih.gov/pubmed/35392437
http://dx.doi.org/10.1177/20406207221085202
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author Hart, Daniel P.
Matino, Davide
Astermark, Jan
Dolan, Gerard
d’Oiron, Roseline
Hermans, Cédric
Jiménez-Yuste, Victor
Linares, Adriana
Matsushita, Tadashi
McRae, Simon
Ozelo, Margareth C.
Platton, Sean
Stafford, Darrel
Sidonio, Robert F.
Tiede, Andreas
author_facet Hart, Daniel P.
Matino, Davide
Astermark, Jan
Dolan, Gerard
d’Oiron, Roseline
Hermans, Cédric
Jiménez-Yuste, Victor
Linares, Adriana
Matsushita, Tadashi
McRae, Simon
Ozelo, Margareth C.
Platton, Sean
Stafford, Darrel
Sidonio, Robert F.
Tiede, Andreas
author_sort Hart, Daniel P.
collection PubMed
description Haemophilia B is a rare X-linked genetic deficiency of coagulation factor IX (FIX) that, if untreated, can cause recurrent and disabling bleeding, potentially leading to severe arthropathy and/or life-threatening haemorrhage. Recent decades have brought significant improvements in haemophilia B management, including the advent of recombinant FIX and extended half-life FIX. This therapeutic landscape continues to evolve with several non-factor replacement therapies and gene therapies under investigation. Given the rarity of haemophilia B, the evidence base and clinical experience on which to establish clinical guidelines are relatively sparse and are further challenged by features that are distinct from haemophilia A, precluding extrapolation of existing haemophilia A guidelines. Due to the paucity of formal haemophilia B-specific clinical guidance, an international Author Group was convened to develop a clinical practice framework. The group comprised 15 haematology specialists from Europe, Australia, Japan, Latin America and North America, covering adult and paediatric haematology, laboratory medicine and biomedical science. A hybrid approach combining a systematic review of haemophilia B literature with discussion of clinical experience utilized a modified Delphi format to develop a comprehensive set of clinical recommendations. This approach resulted in 29 recommendations for the clinical management of haemophilia B across five topics, including product treatment choice, therapeutic agent laboratory monitoring, pharmacokinetics considerations, inhibitor management and preparing for gene therapy. It is anticipated that this clinical practice framework will complement existing guidelines in the management of people with haemophilia B in routine clinical practice and could be adapted and applied across different regions and countries.
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spelling pubmed-89804302022-04-06 International consensus recommendations on the management of people with haemophilia B Hart, Daniel P. Matino, Davide Astermark, Jan Dolan, Gerard d’Oiron, Roseline Hermans, Cédric Jiménez-Yuste, Victor Linares, Adriana Matsushita, Tadashi McRae, Simon Ozelo, Margareth C. Platton, Sean Stafford, Darrel Sidonio, Robert F. Tiede, Andreas Ther Adv Hematol Review Haemophilia B is a rare X-linked genetic deficiency of coagulation factor IX (FIX) that, if untreated, can cause recurrent and disabling bleeding, potentially leading to severe arthropathy and/or life-threatening haemorrhage. Recent decades have brought significant improvements in haemophilia B management, including the advent of recombinant FIX and extended half-life FIX. This therapeutic landscape continues to evolve with several non-factor replacement therapies and gene therapies under investigation. Given the rarity of haemophilia B, the evidence base and clinical experience on which to establish clinical guidelines are relatively sparse and are further challenged by features that are distinct from haemophilia A, precluding extrapolation of existing haemophilia A guidelines. Due to the paucity of formal haemophilia B-specific clinical guidance, an international Author Group was convened to develop a clinical practice framework. The group comprised 15 haematology specialists from Europe, Australia, Japan, Latin America and North America, covering adult and paediatric haematology, laboratory medicine and biomedical science. A hybrid approach combining a systematic review of haemophilia B literature with discussion of clinical experience utilized a modified Delphi format to develop a comprehensive set of clinical recommendations. This approach resulted in 29 recommendations for the clinical management of haemophilia B across five topics, including product treatment choice, therapeutic agent laboratory monitoring, pharmacokinetics considerations, inhibitor management and preparing for gene therapy. It is anticipated that this clinical practice framework will complement existing guidelines in the management of people with haemophilia B in routine clinical practice and could be adapted and applied across different regions and countries. SAGE Publications 2022-04-02 /pmc/articles/PMC8980430/ /pubmed/35392437 http://dx.doi.org/10.1177/20406207221085202 Text en © The Author(s), 2022 https://creativecommons.org/licenses/by-nc/4.0/This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (https://creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access page (https://us.sagepub.com/en-us/nam/open-access-at-sage).
spellingShingle Review
Hart, Daniel P.
Matino, Davide
Astermark, Jan
Dolan, Gerard
d’Oiron, Roseline
Hermans, Cédric
Jiménez-Yuste, Victor
Linares, Adriana
Matsushita, Tadashi
McRae, Simon
Ozelo, Margareth C.
Platton, Sean
Stafford, Darrel
Sidonio, Robert F.
Tiede, Andreas
International consensus recommendations on the management of people with haemophilia B
title International consensus recommendations on the management of people with haemophilia B
title_full International consensus recommendations on the management of people with haemophilia B
title_fullStr International consensus recommendations on the management of people with haemophilia B
title_full_unstemmed International consensus recommendations on the management of people with haemophilia B
title_short International consensus recommendations on the management of people with haemophilia B
title_sort international consensus recommendations on the management of people with haemophilia b
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8980430/
https://www.ncbi.nlm.nih.gov/pubmed/35392437
http://dx.doi.org/10.1177/20406207221085202
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