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Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin (R)): a prospective study of 50 patients

Borel-Derlon, A.; Federici, A. B.; Roussel-Robert, V.; Goudemand, J.; Lee, C. A.; Scharrer, I.; Rothschild, C.; Berntorp, Erik LU ; Henriet, C. and Tellier, Z., et al. (2007) In Journal of Thrombosis and Haemostasis 5(6). p.1115-1124
Abstract
Background and objectives: A plasma-derived von Willebrand factor (VWF) concentrate with low factor VIII (FVIII) content was specifically developed to treat von Willebrand disease (VWD). Efficacy and safety were investigated by merging the results of two comparable protocols conducted prospectively in 5 European and 12 French centers. Methods and results: Fifty patients with clinically severe VWD (72% had VWF ristocetin cofactor activity less than 10 IU dL(-1) and 46% had FVIII < 20 IU dL(-1)) were treated with the concentrate as the only therapy, except for clinical situations requiring a priming dose of FVIII to rapidly correct an intrinsic coagulation defect. A total of 139 spontaneous bleeding episodes were treated; only 53 (38%)... (More)
Background and objectives: A plasma-derived von Willebrand factor (VWF) concentrate with low factor VIII (FVIII) content was specifically developed to treat von Willebrand disease (VWD). Efficacy and safety were investigated by merging the results of two comparable protocols conducted prospectively in 5 European and 12 French centers. Methods and results: Fifty patients with clinically severe VWD (72% had VWF ristocetin cofactor activity less than 10 IU dL(-1) and 46% had FVIII < 20 IU dL(-1)) were treated with the concentrate as the only therapy, except for clinical situations requiring a priming dose of FVIII to rapidly correct an intrinsic coagulation defect. A total of 139 spontaneous bleeding episodes were treated; only 53 (38%) needed a concomitant FVIII dose. Outcome was excellent or good in 89% of the episodes. Forty-four patients underwent 108 surgical or invasive procedures. Outcome was excellent or good in 95 scheduled procedures (only VWF was infused) and 13 emergency procedures (a priming FVIII dose was co-administered with the first VWF infusion). There were no thrombotic complications and none of the 18 patients with type 3 VWD developed anti-VWF or anti-FVIII antibodies. Conclusions. This concentrate safely and effectively provides hemostasis in patients with clinically severe VWD. (Less)
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Contribution to journal
publication status
published
subject
keywords
von Willebrand disease, von Willebrand factor, concentrate
in
Journal of Thrombosis and Haemostasis
volume
5
issue
6
pages
1115 - 1124
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • wos:000246795800004
  • scopus:34250169072
ISSN
1538-7933
DOI
10.1111/j.1538-7836.2007.02562.x
language
English
LU publication?
yes
id
6fd08d0f-4331-473d-a792-53d80b44db15 (old id 662539)
date added to LUP
2007-12-11 14:20:56
date last changed
2017-07-02 03:42:19
@article{6fd08d0f-4331-473d-a792-53d80b44db15,
  abstract     = {Background and objectives: A plasma-derived von Willebrand factor (VWF) concentrate with low factor VIII (FVIII) content was specifically developed to treat von Willebrand disease (VWD). Efficacy and safety were investigated by merging the results of two comparable protocols conducted prospectively in 5 European and 12 French centers. Methods and results: Fifty patients with clinically severe VWD (72% had VWF ristocetin cofactor activity less than 10 IU dL(-1) and 46% had FVIII &lt; 20 IU dL(-1)) were treated with the concentrate as the only therapy, except for clinical situations requiring a priming dose of FVIII to rapidly correct an intrinsic coagulation defect. A total of 139 spontaneous bleeding episodes were treated; only 53 (38%) needed a concomitant FVIII dose. Outcome was excellent or good in 89% of the episodes. Forty-four patients underwent 108 surgical or invasive procedures. Outcome was excellent or good in 95 scheduled procedures (only VWF was infused) and 13 emergency procedures (a priming FVIII dose was co-administered with the first VWF infusion). There were no thrombotic complications and none of the 18 patients with type 3 VWD developed anti-VWF or anti-FVIII antibodies. Conclusions. This concentrate safely and effectively provides hemostasis in patients with clinically severe VWD.},
  author       = {Borel-Derlon, A. and Federici, A. B. and Roussel-Robert, V. and Goudemand, J. and Lee, C. A. and Scharrer, I. and Rothschild, C. and Berntorp, Erik and Henriet, C. and Tellier, Z. and Bridey, F. and Mannucci, P. M.},
  issn         = {1538-7933},
  keyword      = {von Willebrand disease,von Willebrand factor,concentrate},
  language     = {eng},
  number       = {6},
  pages        = {1115--1124},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {Journal of Thrombosis and Haemostasis},
  title        = {Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin (R)): a prospective study of 50 patients},
  url          = {http://dx.doi.org/10.1111/j.1538-7836.2007.02562.x},
  volume       = {5},
  year         = {2007},
}