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Strategies for reducing inhibitor formation in severe haemophilia

Tunstall, Oliver and Astermark, Jan LU (2015) In European Journal of Haematology 94. p.45-50
Abstract
The greatest barrier to successful haemophilia A care in resource-rich countries is the development of inhibitors to therapeutic factor VIII. Children with inhibitors suffer through increased bleeding and joint damage as well as frequent venepuncture. Costs associated with inhibitors are beyond many healthcare systems. Over the last two decades, there has been no improvement in our ability to reduce inhibitor development. Current strategies based on early prophylaxis and avoidance of immunological danger signals have given rise to conflicting results. Registry data suggest an increasing problem. Our understanding of the immunological systems involved in inhibitor formation should inform efforts to prevent inhibitors. Great efforts with... (More)
The greatest barrier to successful haemophilia A care in resource-rich countries is the development of inhibitors to therapeutic factor VIII. Children with inhibitors suffer through increased bleeding and joint damage as well as frequent venepuncture. Costs associated with inhibitors are beyond many healthcare systems. Over the last two decades, there has been no improvement in our ability to reduce inhibitor development. Current strategies based on early prophylaxis and avoidance of immunological danger signals have given rise to conflicting results. Registry data suggest an increasing problem. Our understanding of the immunological systems involved in inhibitor formation should inform efforts to prevent inhibitors. Great efforts with mouse models are being undertaken in this field. However, there is no guarantee of the availability of any new agents arising from experimental work in the near future. Global immunomodulatory agents may be one solution. Compounds with a long history of use in inflammatory conditions have demonstrated efficacy in preventing antibodies to protein therapeutics both in animal models and in humans. As these compounds have a long history of use in humans, including babies, it may be that these agents offer an option for reducing inhibitor formation in previously untreated patients. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
severe haemophilia, inhibitors, children, genetics, prophylaxis, immunomodulatory agents
in
European Journal of Haematology
volume
94
pages
45 - 50
publisher
Wiley-Blackwell
external identifiers
  • wos:000347371100009
  • scopus:84920265229
ISSN
1600-0609
DOI
10.1111/ejh.12501
language
English
LU publication?
yes
id
360cbe29-d98a-4453-aaf8-ba4f58dacdc4 (old id 5076014)
date added to LUP
2015-02-28 09:04:09
date last changed
2017-03-05 03:18:06
@article{360cbe29-d98a-4453-aaf8-ba4f58dacdc4,
  abstract     = {The greatest barrier to successful haemophilia A care in resource-rich countries is the development of inhibitors to therapeutic factor VIII. Children with inhibitors suffer through increased bleeding and joint damage as well as frequent venepuncture. Costs associated with inhibitors are beyond many healthcare systems. Over the last two decades, there has been no improvement in our ability to reduce inhibitor development. Current strategies based on early prophylaxis and avoidance of immunological danger signals have given rise to conflicting results. Registry data suggest an increasing problem. Our understanding of the immunological systems involved in inhibitor formation should inform efforts to prevent inhibitors. Great efforts with mouse models are being undertaken in this field. However, there is no guarantee of the availability of any new agents arising from experimental work in the near future. Global immunomodulatory agents may be one solution. Compounds with a long history of use in inflammatory conditions have demonstrated efficacy in preventing antibodies to protein therapeutics both in animal models and in humans. As these compounds have a long history of use in humans, including babies, it may be that these agents offer an option for reducing inhibitor formation in previously untreated patients.},
  author       = {Tunstall, Oliver and Astermark, Jan},
  issn         = {1600-0609},
  keyword      = {severe haemophilia,inhibitors,children,genetics,prophylaxis,immunomodulatory agents},
  language     = {eng},
  pages        = {45--50},
  publisher    = {Wiley-Blackwell},
  series       = {European Journal of Haematology},
  title        = {Strategies for reducing inhibitor formation in severe haemophilia},
  url          = {http://dx.doi.org/10.1111/ejh.12501},
  volume       = {94},
  year         = {2015},
}