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

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.The Hopscotch Will II study examined the treatment of VWD in hospitals and compared the use of pdVWF and rVWF.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.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, <i>p</i> = 0.2025).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.

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Publication Details

Journal
Seminars in Thrombosis and Hemostasis
Published
2026-01-13
DOI
https://doi.org/10.1055/a-2779-9176
Primary Topic
Platelet Disorders and Treatments
Type
article
Field-Weighted Citation Impact
0.00

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article

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

Philippe Beurrier, Marc Trossaërt, Catherine Ternisien, Valérie Horvais et al.
Seminars in Thrombosis and Hemostasis
Platelet Disorders and Treatments
article

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

Philippe Beurrier, Marc Trossaërt, Catherine Ternisien, Valérie Horvais, Nicolas Drillaud, Marc Fouassier, Johann Rose, Vincent Cussac, Benoît Guillet, Julien Denis-Le-Sève, Brigitte Pan-Petesch, Sophie Bayart, Antoine Babuty
article en

Abstract

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.The Hopscotch Will II study examined the treatment of VWD in hospitals and compared the use of pdVWF and rVWF.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.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, <i>p</i> = 0.2025).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.

Seminars in Thrombosis and Hemostasis
Inserm (FR), Institut de Recherche en Santé, Environnement et Travail (FR), Centre Hospitalier Universitaire d'Angers (FR), Centre Hospitalier du Mans (FR), Centre d'Investigation Clinique de Nantes (FR), Université de Rennes (FR), Nantes Université (FR)
Baxalta
Good health and well-being
Openalex Percentile: Top 90%
Platelet Disorders and Treatments
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