# Management of von Willebrand disease with a factor VIII-poor von Willebrand factor concentrate: Results from a prospective observational post-marketing study

Nathalie Itzhar-baikian, Annie Borel-derlon, Jenny Goudemand, Françoise Bridey, Segolene Claeyssens, Nathalie Itzhar-Baikian, Annie Harroche, Dominique Desprez, Claude Negrier, Pierre Chamouni, et al.

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Nathalie Itzhar-baikian, Annie Borel-derlon, Jenny Goudemand, Françoise Bridey, Segolene Claeyssens, et al. Management of von Willebrand disease with a factor VIII-poor von Willebrand factor concentrate: Results from a prospective observational post-marketing study. Journal of Thrombosis and Haemostasis, 2020, 18 (8), pp.1922-1933. 10.1111/jth.14928. hal-03143538

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ARTICLE

Management of von Willebrand disease with a factor VIII-poor von
Willebrand factor concentrate: Results from a prospective
observational post-marketing study

Jenny Goudemand, Françoise Bridey, Ségolène Claeyssens, Nathalie
Itzhar-Baïkian, Annie Harroche, Dominique Desprez, Claude Négrier,
Pierre Chamouni, Hervé Chambost, Céline Henriet, Sophie Susen, Annie
Borel-Derlon
format_quote CITE
<https://onlinelibrary.wiley.com/action/showCitFormats?doi=10.1111%2Fjth.
14928&mobileUi=0>

© 2020 The Authors. /Journal of Thrombosis and Haemostasis/ published by
Wiley Periodicals LLC on behalf of International Society on Thrombosis
and Haemostasis
https://doi.org/10.1111/jth.14928 <https://doi.org/10.1111/jth.14928>
Published inJournal of Thrombosis and Haemostasis
ISSN1538-7933
eISSN1538-7836

Received 2 December 2019

Accepted 14 May 2020

Published 4 August 2020

Volume18

Issue8

Pages1922 - 1933

Abstract

Background

A triple-secured plasma-derived von Willebrand factor (pdVWF) almost devoid of factor VIII (FVIII):WILFACTIN^®, was approved in France in 2003, and then in other countries for the treatment of patients with von Willebrand disease (VWD).

Objective
To investigate long-term safety and efficacy of the product in real-life
over the first 5 post-approval years.

## Patients/Methods

This prospective, observational, national post-marketing study (PMS)
enrolled patients of all ages and VWD types. Patients were observed for
up to 3 years and treated for one or more occasions. Efficacy was
assessed for each major event. Breakthrough bleeding rate 3 days
post-infusion and annualized bleeding rate (ABR) were also evaluated for
long-term prophylaxis.

## Results

Overall, 155 of 174 patients enrolled from 31 centers were eligible for
efficacy assessment. Most patients (76.8%) were severely affected
(VWF:RCo ≤ 15 IU/dL). They were treated for 743 bleeds and 140 surgeries
including childbirth. Efficacy outcomes were excellent/good for 98.2% of
56 major surgeries and 94.0% of 67 major bleeds. Approximately 75% of
49 major mucosal bleeds were effectively managed without FVIII
co-administration. In 32 patients receiving prophylaxis, breakthrough
bleeding occurred in 1.5% of infusions and median ABR was 1.0 for 20
patients treated ≥ 12 months. Excellent tolerability was confirmed with
no safety concerns. No thrombotic events were observed.

## Conclusions

Results from this PMS increase the clinical experience of a FVIII-poor
pdVWF in patients of all ages and VWD types including those with
thrombotic risk factors and emphasize that giving FVIII is not always
mandatory to effectively treat patients with severe VWD.

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