Prophylaxis in severe forms of von Willebrand's disease

Results from the von Willebrand Disease Prophylaxis Network (VWD PN)

T. C. Abshire, A. B. Federici, M. T. Alvárez, J. Bowen, M. D. Carcao, J. Cox Gill, N. S. Key, P. A. Kouides, K. Kurnik, A. E. Lail, F. W G Leebeek, M. Makris, P. M. Mannucci, R. Winikoff, E. Berntorp

Research output: Contribution to journalArticle

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Abstract

The bleeding patterns of severe von Willebrand's disease (VWD) adversely affect quality of life, and may be life threatening. There is a presumed role for prophylaxis with VWF-containing concentrates, but data are scarce. The von Willebrand Disease Prophylaxis Network (VWD PN) was formed to investigate the role of prophylaxis in clinically severe VWD that is not responsive to other treatment(s).Using a retrospective design, the effect of prophylaxis was studied. Availability of records to document, or reliably assess, the type and frequency of bleeding episodes prior to, and after, the initiation of prophylaxis was required. Annualized bleeding rates were calculated for the period prior to prophylaxis, during prophylaxis and by primary bleeding indication defined as the site accounting for more than half of all bleeding symptoms. The Wilcoxon signed-rank test of differences in the medians was used. Sixty-one subjects from 20 centres in 10 countries were enrolled. Data for 59 were used in the analysis. The median age at onset of prophylaxis was 22.4 years. Type 3 VWD accounted for the largest number (N = 34, 57.6%). Differences in bleeding rates within individuals during compared with before prophylaxis were significant for the total group (P <0.0001), and for those with primary bleeding indications of epistaxis (P = 0.0005), joint bleeding (P = 0.002) and GI bleeding (P = 0.001). The effect of prophylaxis was similar among those age <18 years and those ≥18. One person developed an inhibitor during treatment. We conclude that prophylactic treatment of VWD is efficacious.

Original languageEnglish
Pages (from-to)76-81
Number of pages6
JournalHaemophilia
Volume19
Issue number1
DOIs
Publication statusPublished - Jan 2013

Fingerprint

Type 3 Von Willebrand's Disease
von Willebrand Diseases
Hemorrhage
Epistaxis
Nonparametric Statistics
Age of Onset
Joints

Keywords

  • Bleeding rate
  • Epistaxis
  • Gastrointestinal bleeding
  • Joint bleeding
  • Prophylaxis
  • Severe VWD

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Abshire, T. C., Federici, A. B., Alvárez, M. T., Bowen, J., Carcao, M. D., Cox Gill, J., ... Berntorp, E. (2013). Prophylaxis in severe forms of von Willebrand's disease: Results from the von Willebrand Disease Prophylaxis Network (VWD PN). Haemophilia, 19(1), 76-81. https://doi.org/10.1111/j.1365-2516.2012.02916.x

Prophylaxis in severe forms of von Willebrand's disease : Results from the von Willebrand Disease Prophylaxis Network (VWD PN). / Abshire, T. C.; Federici, A. B.; Alvárez, M. T.; Bowen, J.; Carcao, M. D.; Cox Gill, J.; Key, N. S.; Kouides, P. A.; Kurnik, K.; Lail, A. E.; Leebeek, F. W G; Makris, M.; Mannucci, P. M.; Winikoff, R.; Berntorp, E.

In: Haemophilia, Vol. 19, No. 1, 01.2013, p. 76-81.

Research output: Contribution to journalArticle

Abshire, TC, Federici, AB, Alvárez, MT, Bowen, J, Carcao, MD, Cox Gill, J, Key, NS, Kouides, PA, Kurnik, K, Lail, AE, Leebeek, FWG, Makris, M, Mannucci, PM, Winikoff, R & Berntorp, E 2013, 'Prophylaxis in severe forms of von Willebrand's disease: Results from the von Willebrand Disease Prophylaxis Network (VWD PN)', Haemophilia, vol. 19, no. 1, pp. 76-81. https://doi.org/10.1111/j.1365-2516.2012.02916.x
Abshire, T. C. ; Federici, A. B. ; Alvárez, M. T. ; Bowen, J. ; Carcao, M. D. ; Cox Gill, J. ; Key, N. S. ; Kouides, P. A. ; Kurnik, K. ; Lail, A. E. ; Leebeek, F. W G ; Makris, M. ; Mannucci, P. M. ; Winikoff, R. ; Berntorp, E. / Prophylaxis in severe forms of von Willebrand's disease : Results from the von Willebrand Disease Prophylaxis Network (VWD PN). In: Haemophilia. 2013 ; Vol. 19, No. 1. pp. 76-81.
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