Abstract
Background
Von Willebrand disease (VWD) is the most common inherited bleeding disorder. However, studies of hospitalisation patterns with replacement treatment are scarce.
Objectives
The aim of this study was to investigate the current therapeutic management of VWD and determine the key drivers of coagulation factor uses in patients during hospitalisation.
Methods
Hopscotch-WILL was a multi-centric retrospective study conducted over a 48-month period in any patients with VWD. The data were collected from the BERHLINGO Research Database and the French Hospital database.
Results
A total of 988 patients were included; 153 patients (15%) were hospitalised during 293 stays requiring treatment with von Willebrand factor (VWF) concentrates—pure or in association with Factor VIII (FVIII). Their median basal concentrations of VWF and FVIII were significantly lower than in untreated patients: VWF antigen < 30 IU/dL, VWF activity < 20 IU/dL and FVIII:C < 40 IU/dL. The median (interquartile range) concentrate consumption was similar between highly purified VWF or VWF combined with FVIII (72 [110] vs 57 [89] IU/kg/stay, p = 0.154). The use of VWF was highly heterogeneous by VWD type; type 3 had a particularly high impact on VWF consumption in non-surgical situations. The main admissions were for ear/nose/throat, hepato-gastroenterology, and trauma/orthopaedic conditions, besides gynaecological-obstetric causes in women.
Conclusions
The use of VWF concentrates is mostly influenced by low basal levels of VWF and FVIII, but also by VWD type or the cause for hospitalisation. These results could inform future studies of newly released recombinant VWF.
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Acknowledgements
The authors thank the patients and their families for participating in this study. This study was conducted within the French network of University Hospitals HUGO (Hôpitaux Universitaires du Grand Ouest). The authors would like to thank Ms Jill Rupnow (Kokopelli SARL, Paris, France) for providing medical writing services; these services were funded through the study budget. Lastly, the authors thank the members of the BERHLINGO consortium for their daily involvement in the collection of high-quality data and for the attention they devoted to this research project. The BERHLINGO consortium: Sabrina Cochennec, Magalie Cornec, Guillaume Drugmanne, Hubert Galinat, Isabelle Gouin, Estelle Leroy, Fabienne Nedelec-Gac.
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This study was funded by Baxalta GmbH, a wholly subsidiary of Takeda Pharmaceutical Company Limited, as part of an investigator-initiated research project (IIR-FXX-002472). Baxalta GmbH played no role in the design and conduct of the study/collection, management, analysis, or interpretation of the data/preparation, writing, review, or approval of the manuscript.
Conflicts of Interest
Valérie Horvais, Philippe Beurrier, Vincent Cussac, Brigitte Pan-Petesch, Solène Schirr-Bonnans, Johann Rose, Sophie Bayart, Catherine Ternisien, Marc Fouassier, Marianne Sigaud, Antoine Babuty and Nicolas Drillaud declare that they have no conflict of interest. Benoît Guillet received research grants from CSL Behring and consulting fees from CSL Behring/LFB/Takeda. Marc Trossaërt received funding from Baxalta GmbH for the Hopscotch Will I study.
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The data underlying the findings of this study are not openly available because they are sensitive. They can be obtained from the corresponding author upon reasonable request. The data are stored in a controlled access facility at Nantes University Hospital.
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The Institutional Ethics Committee approved the study protocol on 3 April 2019 (Groupe Nantais d'Ethique dans le Domaine de la Santé, GNEDS).
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The patient's non-objection was required for inclusion in the study. No written consent was required.
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Author Contributions
Valérie Horvais, Solène Schirr-Bonnans and Marc Trossaërt contributed to the conception and design of the study. All of the authors contributed to the preparation of the materials and collection of the data. The analyses were performed by Valérie Horvais and Solène Schirr-Bonnans. The first draft of the manuscript was written by Valérie Horvais and all authors commented on all intermediary versions of the manuscript. All authors read and approved the final manuscript.
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The members of the BERHLINGO consortium involved in the study are listed in the “Acknowledgements" section.
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Horvais, V., Beurrier, P., Cussac, V. et al. Key Drivers of Coagulation Factor Use in Von Willebrand Disease During Hospitalization: An Overview of the French BERHLINGO Cohort. Clin Drug Investig 44, 35–49 (2024). https://doi.org/10.1007/s40261-023-01323-1
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DOI: https://doi.org/10.1007/s40261-023-01323-1