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Haemophilia B : Where are we now and what does the future hold?

Dolan, Gerry; Benson, Gary; Duffy, Anne; Hermans, Cedric; Jiménez-Yuste, Victor; Lambert, Thierry; Ljung, Rolf LU ; Morfini, Massimo and Zupančić Šalek, Silva (2017) In Blood Reviews
Abstract

Research has been lacking on the natural history, complications, and treatment of haemophilia B, which is less common than haemophilia A and was recognized as a distinct clinical entity in 1947. Although the two diseases share the same clinical manifestations, they differ in causative mutation, risk of inhibitor development, and patient quality of life. Frequently debated is whether haemophilia B is as clinically severe as haemophilia A, with much of the published data on overall and haemophilia-specific health outcomes suggesting that haemophilia B may have a less severe clinical phenotype. However, although fewer haemophilia B than haemophilia A patients appear to experience bleeding, bleeds are just as severe. We review... (More)

Research has been lacking on the natural history, complications, and treatment of haemophilia B, which is less common than haemophilia A and was recognized as a distinct clinical entity in 1947. Although the two diseases share the same clinical manifestations, they differ in causative mutation, risk of inhibitor development, and patient quality of life. Frequently debated is whether haemophilia B is as clinically severe as haemophilia A, with much of the published data on overall and haemophilia-specific health outcomes suggesting that haemophilia B may have a less severe clinical phenotype. However, although fewer haemophilia B than haemophilia A patients appear to experience bleeding, bleeds are just as severe. We review distinguishing characteristics of haemophilia B and its treatment, including management strategies for neonates, therapeutic approaches for patients who develop inhibitors, pharmacokinetics of factor IX concentrates administered as replacement therapy, and potential future treatments.

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author
organization
publishing date
type
Contribution to journal
publication status
epub
subject
keywords
FIX concentrates, Gene therapy, Haemophilia B, Inhibitors, Pharmacokinetics, Prophylaxis
in
Blood Reviews
publisher
Churchill Livingstone
external identifiers
  • scopus:85027701911
ISSN
0268-960X
DOI
10.1016/j.blre.2017.08.007
language
English
LU publication?
yes
id
7bd6e707-8fa8-4b89-95c0-4e28acfdc421
date added to LUP
2017-09-04 16:21:41
date last changed
2017-11-16 15:07:01
@article{7bd6e707-8fa8-4b89-95c0-4e28acfdc421,
  abstract     = {<p>Research has been lacking on the natural history, complications, and treatment of haemophilia B, which is less common than haemophilia A and was recognized as a distinct clinical entity in 1947. Although the two diseases share the same clinical manifestations, they differ in causative mutation, risk of inhibitor development, and patient quality of life. Frequently debated is whether haemophilia B is as clinically severe as haemophilia A, with much of the published data on overall and haemophilia-specific health outcomes suggesting that haemophilia B may have a less severe clinical phenotype. However, although fewer haemophilia B than haemophilia A patients appear to experience bleeding, bleeds are just as severe. We review distinguishing characteristics of haemophilia B and its treatment, including management strategies for neonates, therapeutic approaches for patients who develop inhibitors, pharmacokinetics of factor IX concentrates administered as replacement therapy, and potential future treatments.</p>},
  author       = {Dolan, Gerry and Benson, Gary and Duffy, Anne and Hermans, Cedric and Jiménez-Yuste, Victor and Lambert, Thierry and Ljung, Rolf and Morfini, Massimo and Zupančić Šalek, Silva},
  issn         = {0268-960X},
  keyword      = {FIX concentrates,Gene therapy,Haemophilia B,Inhibitors,Pharmacokinetics,Prophylaxis},
  language     = {eng},
  publisher    = {Churchill Livingstone},
  series       = {Blood Reviews},
  title        = {Haemophilia B : Where are we now and what does the future hold?},
  url          = {http://dx.doi.org/10.1016/j.blre.2017.08.007},
  year         = {2017},
}