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The current status of prophylactic replacement therapy in children and adults with haemophilia.

Ljung, Rolf LU and Gretenkort, Nadine LU (2015) In British Journal of Haematology 169(6). p.777-786
Abstract
Initiating prophylactic treatment at an early age is considered to be the optimal form of therapy for a child with haemophilia A or B. The pioneering long term experiences of prophylactic treatment from Sweden and The Netherlands demonstrated the benefit of prophylaxis in retrospective and observational studies. Decades later, these benefits were confirmed in a randomized controlled study in USA. We review the current status of prophylactic replacement therapy of haemophilia in children, adolescents, adults and the elderly. Prophylaxis should begin at an early age and there are arguments for continuing it into adulthood. The dose of prophylaxis is dependent on the goal of treatment, economic resources and venous access and should be... (More)
Initiating prophylactic treatment at an early age is considered to be the optimal form of therapy for a child with haemophilia A or B. The pioneering long term experiences of prophylactic treatment from Sweden and The Netherlands demonstrated the benefit of prophylaxis in retrospective and observational studies. Decades later, these benefits were confirmed in a randomized controlled study in USA. We review the current status of prophylactic replacement therapy of haemophilia in children, adolescents, adults and the elderly. Prophylaxis should begin at an early age and there are arguments for continuing it into adulthood. The dose of prophylaxis is dependent on the goal of treatment, economic resources and venous access and should be tailored individually. Starting the first exposures to clotting factor concentrates as prophylactic treatment, instead of on-demand in response to a bleed, may decrease the frequency of inhibitors in patients with haemophilia A. Novel longer-acting products are being introduced that could be helpful for patients with difficult venous access and enable higher trough levels. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
British Journal of Haematology
volume
169
issue
6
pages
777 - 786
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • pmid:25819695
  • wos:000355657300005
  • scopus:84930268161
ISSN
0007-1048
DOI
10.1111/bjh.13365
language
English
LU publication?
yes
id
f3562735-2cc8-4264-84eb-9dab2261d999 (old id 5257346)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/25819695?dopt=Abstract
date added to LUP
2015-04-04 16:35:39
date last changed
2017-11-05 03:24:32
@article{f3562735-2cc8-4264-84eb-9dab2261d999,
  abstract     = {Initiating prophylactic treatment at an early age is considered to be the optimal form of therapy for a child with haemophilia A or B. The pioneering long term experiences of prophylactic treatment from Sweden and The Netherlands demonstrated the benefit of prophylaxis in retrospective and observational studies. Decades later, these benefits were confirmed in a randomized controlled study in USA. We review the current status of prophylactic replacement therapy of haemophilia in children, adolescents, adults and the elderly. Prophylaxis should begin at an early age and there are arguments for continuing it into adulthood. The dose of prophylaxis is dependent on the goal of treatment, economic resources and venous access and should be tailored individually. Starting the first exposures to clotting factor concentrates as prophylactic treatment, instead of on-demand in response to a bleed, may decrease the frequency of inhibitors in patients with haemophilia A. Novel longer-acting products are being introduced that could be helpful for patients with difficult venous access and enable higher trough levels.},
  author       = {Ljung, Rolf and Gretenkort, Nadine},
  issn         = {0007-1048},
  language     = {eng},
  number       = {6},
  pages        = {777--786},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {British Journal of Haematology},
  title        = {The current status of prophylactic replacement therapy in children and adults with haemophilia.},
  url          = {http://dx.doi.org/10.1111/bjh.13365},
  volume       = {169},
  year         = {2015},
}