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CC BY-NC-ND 4.0 · Semin Thromb Hemost
DOI: 10.1055/a-2779-9176
Original Article

Vonicog Alfa versus Plasma-Derived von Willebrand Factor During Hospitalization: Results of an Observational Retrospective Multicenter Study

Authors

  • Valérie Horvais 

    1   Nantes Université, CHU Nantes, Unité d'Investigation Clinique 17, Nantes, France

  • Catherine Ternisien

    2   Nantes Université, CHU Nantes, Centre de Ressources et de Compétences des Maladies Hémorragiques Constitutionnelles, Nantes, France

  • Julien Denis-Le-Sève 

    3   CHU Angers, Centre de Traitement des Maladies Hémorragiques Constitutionnelles, Angers, France

  • Philippe Beurrier 

    3   CHU Angers, Centre de Traitement des Maladies Hémorragiques Constitutionnelles, Angers, France

  • Marc Fouassier 

    2   Nantes Université, CHU Nantes, Centre de Ressources et de Compétences des Maladies Hémorragiques Constitutionnelles, Nantes, France

  • Antoine Babuty 

    2   Nantes Université, CHU Nantes, Centre de Ressources et de Compétences des Maladies Hémorragiques Constitutionnelles, Nantes, France

  • Nicolas Drillaud 

    2   Nantes Université, CHU Nantes, Centre de Ressources et de Compétences des Maladies Hémorragiques Constitutionnelles, Nantes, France

  • Brigitte Pan-Petesch 

    4   CHU Brest, Service Hématologie Clinique Hémostase, Brest, France

  • Johann Rose 

    5   CH Le Mans, Centre de Ressources et de Compétences des Maladies Hémorragiques Constitutionnelles, Le Mans, France

  • Vincent Cussac 

    5   CH Le Mans, Centre de Ressources et de Compétences des Maladies Hémorragiques Constitutionnelles, Le Mans, France

  • Sophie Bayart 

    6   CHU Rennes, Centre de Ressources et de Compétences des Maladies Hémorragiques Constitutionnelles, Rennes, France

  • Benoît Guillet 

    6   CHU Rennes, Centre de Ressources et de Compétences des Maladies Hémorragiques Constitutionnelles, Rennes, France
    7   Inserm, Rennes Université, EHESP, IRSET (Institut de recherche en santé, environnement et travail), UMR_S 1085, Rennes, France

  • Marc Trossaërt 

    2   Nantes Université, CHU Nantes, Centre de Ressources et de Compétences des Maladies Hémorragiques Constitutionnelles, Nantes, France

Funding Information This study was funded by Baxalta GmbH, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, as part of an investigator-initiated research project (IISR-2021-200066/Sponsor: Nantes University Hospital). Baxalta GmbH played no role in the design and conduct of the study, the collection, management, analysis, or interpretation of the data, or the preparation, writing, review, or approval of the manuscript.
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Abstract

Background

Available products for the treatment of von Willebrand disease (VWD) now include a new recombinant von Willebrand factor (rVWF) in addition to plasma-derived concentrates (pdVWF). However, these two therapies have never been compared directly in either preclinical studies or real-world inpatient settings.

Objective

The Hopscotch Will II study examined the treatment of VWD in hospitals and compared the use of pdVWF and rVWF.

Methods

Five Rare Bleeding Diseases Centers in Western France retrospectively included patients with VWD over a 48-month period. The data was collected from the BERHLINGO Research Database as well as the French Hospital database, which contains medical information from patient records.

Results

Of the 866 patients evaluated in the study, 285 underwent 648 hospitalizations; 126 adult patients (VWD type 3 excluded) were given VWF concentrates during 249 of those hospital stays. rVWF was used in 61% of the cases. The majority of the hospitalizations were motivated by cutaneous-mucosal symptoms in gastroenterology, stomatology, gynecology, and obstetrics. Consumption of rVWF was lower, though the difference in total VWF consumption per stay or per patient per year was not significant: 51 (57)/34 (30) IU/kg/patient/year for pdVWF versus 40 (47)/27 (26) for rVWF (mean [SD]/median [IQR], Wilcoxon rank sum test, p = 0.2025).

Conclusion

rVWF was used in similar patient profiles and for identical procedures, but the cost of treatment with rVWF was significantly lower, regardless of whether or not FVIII was added.

Keywords

bleeding - hospitalization - inpatients - von Willebrand disease - von Willebrand factor

Data Availability Statement

The Nantes University Hospital does not plan to share data supporting the results reported in this article.


Contributors' Statement

All authors contributed to the design of the study, the acquisition of data, and the interpretation of data. V.H. and M.T. contributed to the conception of the study and the analysis of data. All authors commented on all intermediary versions of the manuscript (drafting the article or revising it critically for important intellectual content). All authors read and approved the final version of the manuscript to be submitted.


Clinical Trial Registration

The study is registered at www.clinicaltrials.gov under the number NCT04887324 (May 14, 2021).


Supplementary Material



Publication History

Received: 15 July 2025

Accepted: 21 October 2025

Article published online:
13 January 2026

© 2026. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/)

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