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Prophylaxis in von Willebrand disease.

Berntorp, Erik LU (2008) In Haemophilia 14 Suppl 5. p.47-53
Abstract
This paper reviews current issues regarding short-term (i.e. for surgery and invasive procedures) and long-term (i.e. regular infusions to prevent bleeding on a permanent or temporary basis) prophylaxis treatment using replacement concentrates for patients with von Willebrand disease (VWD) who do not respond satisfactorily to desmopressin. The standard treatment of these patients is with factor concentrates containing von Willebrand factor (VWF) and factor VIII (FVIII). When dosing these concentrates, the broad variations in content and quality of VWF as well as the FVIII content in the products should be considered. Peri-operative management strategies will depend on the VWD subtype, baseline VWF and FVIII levels, and size of procedure.... (More)
This paper reviews current issues regarding short-term (i.e. for surgery and invasive procedures) and long-term (i.e. regular infusions to prevent bleeding on a permanent or temporary basis) prophylaxis treatment using replacement concentrates for patients with von Willebrand disease (VWD) who do not respond satisfactorily to desmopressin. The standard treatment of these patients is with factor concentrates containing von Willebrand factor (VWF) and factor VIII (FVIII). When dosing these concentrates, the broad variations in content and quality of VWF as well as the FVIII content in the products should be considered. Peri-operative management strategies will depend on the VWD subtype, baseline VWF and FVIII levels, and size of procedure. FVIII level and VWF ristocetin cofactor activity may both be used to determine concentrate potency and to monitor treatment. Long-term prophylaxis, which has become a state-of-the-art approach in haemophilia, is not very common in VWD. However, more recent data suggest that a substantial number of VWD patients could benefit from prophylactic treatment with VWF-containing concentrates. For example, 35 Swedish VWD patients who required prophylaxis (mainly because of nose/mouth bleeds and joint bleeds) showed a substantial overall reduction in bleeding episodes and there were no signs of arthropathy in children who began prophylaxis before the age of 5 years since initiation of treatment with Haemate P/Humate-P. Studies of prophylaxis in VWD are urgently needed to develop evidence-based guidelines for this approach; the VWD International Prophylaxis study, for example, has commenced by the VWD Prophylaxis Network. (Less)
Please use this url to cite or link to this publication:
author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Haemophilia
volume
14 Suppl 5
pages
47 - 53
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • wos:000262621300006
  • pmid:18786010
  • scopus:51349109299
ISSN
1351-8216
DOI
10.1111/j.1365-2516.2008.01851.x
language
English
LU publication?
yes
id
b99c3acb-a8ce-4e67-852d-14dbcaad1250 (old id 1243007)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/18786010?dopt=Abstract
date added to LUP
2008-10-07 14:23:32
date last changed
2017-04-09 04:27:56
@article{b99c3acb-a8ce-4e67-852d-14dbcaad1250,
  abstract     = {This paper reviews current issues regarding short-term (i.e. for surgery and invasive procedures) and long-term (i.e. regular infusions to prevent bleeding on a permanent or temporary basis) prophylaxis treatment using replacement concentrates for patients with von Willebrand disease (VWD) who do not respond satisfactorily to desmopressin. The standard treatment of these patients is with factor concentrates containing von Willebrand factor (VWF) and factor VIII (FVIII). When dosing these concentrates, the broad variations in content and quality of VWF as well as the FVIII content in the products should be considered. Peri-operative management strategies will depend on the VWD subtype, baseline VWF and FVIII levels, and size of procedure. FVIII level and VWF ristocetin cofactor activity may both be used to determine concentrate potency and to monitor treatment. Long-term prophylaxis, which has become a state-of-the-art approach in haemophilia, is not very common in VWD. However, more recent data suggest that a substantial number of VWD patients could benefit from prophylactic treatment with VWF-containing concentrates. For example, 35 Swedish VWD patients who required prophylaxis (mainly because of nose/mouth bleeds and joint bleeds) showed a substantial overall reduction in bleeding episodes and there were no signs of arthropathy in children who began prophylaxis before the age of 5 years since initiation of treatment with Haemate P/Humate-P. Studies of prophylaxis in VWD are urgently needed to develop evidence-based guidelines for this approach; the VWD International Prophylaxis study, for example, has commenced by the VWD Prophylaxis Network.},
  author       = {Berntorp, Erik},
  issn         = {1351-8216},
  language     = {eng},
  pages        = {47--53},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {Haemophilia},
  title        = {Prophylaxis in von Willebrand disease.},
  url          = {http://dx.doi.org/10.1111/j.1365-2516.2008.01851.x},
  volume       = {14 Suppl 5},
  year         = {2008},
}