Advanced

Paediatric haemophilia with inhibitors: existing management options, treatment gaps and unmet needs

Santagostino, E.; Morfini, M.; Auerswald, G. K. -H.; Benson, G. M.; Salek, S. Z.; Lambert, T.; Salaj, P.; Jimenez-Yuste, V. and Ljung, Rolf LU (2009) In Haemophilia 15(5). p.983-989
Abstract
Development of inhibitors is a severe complication of haemophilia posing many management challenges. While a long-term goal in inhibitor patients is eradication of inhibitors through immune tolerance induction, bypassing agents such as recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrate (aPCC) are essential for control of bleeding episodes. Paediatric patients with haemophilia and inhibitors are at particular risk of recurrent haemarthroses, and management of these patients should seek to avoid joint damage and support the child's full social and physical development. Current options for management of bleeding complications include on-demand treatment of acute bleeding episodes, secondary prophylaxis to... (More)
Development of inhibitors is a severe complication of haemophilia posing many management challenges. While a long-term goal in inhibitor patients is eradication of inhibitors through immune tolerance induction, bypassing agents such as recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrate (aPCC) are essential for control of bleeding episodes. Paediatric patients with haemophilia and inhibitors are at particular risk of recurrent haemarthroses, and management of these patients should seek to avoid joint damage and support the child's full social and physical development. Current options for management of bleeding complications include on-demand treatment of acute bleeding episodes, secondary prophylaxis to avoid recurrent bleeds and surgery to treat affected joints. There is also a rationale for adopting prophylactic approaches to prevent bleeding in inhibitor patients, allowing this group similar opportunities for protection against arthropathy development as are given to non-inhibitor patients. This paper, based on a roundtable meeting of haematology experts at the first Zurich Haemophilia Forum in May 2008, reviews the current evidence supporting more intense and prophylactic approaches to manage bleeding risk in paediatric haemophilia patients with inhibitors, and highlights the need for investigations of primary prophylaxis in this vulnerable patient group, to support best long-term outcome. (Less)
Please use this url to cite or link to this publication:
author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
paediatric, inhibitors, activated prothrombin complex concentrate, haemophilia, prophylaxis, recombinant activated factor VII
in
Haemophilia
volume
15
issue
5
pages
983 - 989
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • wos:000269367800001
  • scopus:69949096687
ISSN
1351-8216
DOI
10.1111/j.1365-2516.2009.01999.x
language
English
LU publication?
yes
id
f70116c1-c3ad-4f6c-aa15-0a2d87dbb55d (old id 1476316)
date added to LUP
2009-09-29 12:41:01
date last changed
2017-07-23 03:38:05
@article{f70116c1-c3ad-4f6c-aa15-0a2d87dbb55d,
  abstract     = {Development of inhibitors is a severe complication of haemophilia posing many management challenges. While a long-term goal in inhibitor patients is eradication of inhibitors through immune tolerance induction, bypassing agents such as recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrate (aPCC) are essential for control of bleeding episodes. Paediatric patients with haemophilia and inhibitors are at particular risk of recurrent haemarthroses, and management of these patients should seek to avoid joint damage and support the child's full social and physical development. Current options for management of bleeding complications include on-demand treatment of acute bleeding episodes, secondary prophylaxis to avoid recurrent bleeds and surgery to treat affected joints. There is also a rationale for adopting prophylactic approaches to prevent bleeding in inhibitor patients, allowing this group similar opportunities for protection against arthropathy development as are given to non-inhibitor patients. This paper, based on a roundtable meeting of haematology experts at the first Zurich Haemophilia Forum in May 2008, reviews the current evidence supporting more intense and prophylactic approaches to manage bleeding risk in paediatric haemophilia patients with inhibitors, and highlights the need for investigations of primary prophylaxis in this vulnerable patient group, to support best long-term outcome.},
  author       = {Santagostino, E. and Morfini, M. and Auerswald, G. K. -H. and Benson, G. M. and Salek, S. Z. and Lambert, T. and Salaj, P. and Jimenez-Yuste, V. and Ljung, Rolf},
  issn         = {1351-8216},
  keyword      = {paediatric,inhibitors,activated prothrombin complex concentrate,haemophilia,prophylaxis,recombinant activated factor VII},
  language     = {eng},
  number       = {5},
  pages        = {983--989},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {Haemophilia},
  title        = {Paediatric haemophilia with inhibitors: existing management options, treatment gaps and unmet needs},
  url          = {http://dx.doi.org/10.1111/j.1365-2516.2009.01999.x},
  volume       = {15},
  year         = {2009},
}