Document Detail


Prophylaxis in severe forms of von Willebrand's disease: results from the von Willebrand Disease Prophylaxis Network (VWD PN).
MedLine Citation:
PMID:  22823000     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
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.
Authors:
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;
Publication Detail:
Type:  Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't     Date:  2012-07-23
Journal Detail:
Title:  Haemophilia : the official journal of the World Federation of Hemophilia     Volume:  19     ISSN:  1365-2516     ISO Abbreviation:  Haemophilia     Publication Date:  2013 Jan 
Date Detail:
Created Date:  2012-12-12     Completed Date:  2013-05-14     Revised Date:  2013-07-11    
Medline Journal Info:
Nlm Unique ID:  9442916     Medline TA:  Haemophilia     Country:  England    
Other Details:
Languages:  eng     Pagination:  76-81     Citation Subset:  IM    
Copyright Information:
© 2012 Blackwell Publishing Ltd.
Affiliation:
Medical Sciences Institute, BloodCenter of Wisconsin, Milwaukee, WI, USA.
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MeSH Terms
Descriptor/Qualifier:
Adolescent
Adult
Aged
Child
Child, Preschool
Coagulants / therapeutic use*
Drug Administration Schedule
Female
Hemarthrosis / etiology,  prevention & control
Hemorrhage / etiology,  prevention & control*
Humans
Male
Middle Aged
Retrospective Studies
Young Adult
von Willebrand Diseases / complications,  drug therapy*
von Willebrand Factor / therapeutic use*
Grant Support
ID/Acronym/Agency:
UL1 RR031973/RR/NCRR NIH HHS
Chemical
Reg. No./Substance:
0/Coagulants; 0/von Willebrand Factor
Investigator
Investigator/Affiliation:
E Berntorp / ; T Abshire / ; M Alvárez / ; J Astermark / ; J Blatny / ; P Bolton-Maggs / ; L Bowles / ; M Carcao / ; S Crary / ; A B Federici / ; A Geddis / ; P Giardina / ; J Cox Gill / ; K Kavakli / ; C Kempton / ; B Kerlin / ; N Key / ; R Klamroth / ; E Kraut / ; P Kouides / ; K Kurnik / ; A Landorph / ; F Leebeek / ; S Lethagen / ; M Makris / ; P M Mannucci / ; P Mathew / ; D Nugent / ; S Pavord / ; A Shapiro / ; J Wilde / ; L Valentino / ; R Winikoff / ; T Yee /

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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