Key Drivers of Coagulation Factor Use in Von Willebrand Disease During Hospitalization: An Overview of the French BERHLINGO Cohort

Authors: Horvais, V; Beurrier, P; Cussac, V; Pan-Petesch, B; Schirr-Bonnans, S; Rose, J; Bayart, S; Ternisien, C; Fouassier, M; Sigaud, M; Babuty, A; Drillaud, N; Guillet, B; Trossaërt, M.

Affiliations: Nantes Université, CHU Nantes, Unité d’Investigation Clinique 17, 44000, Nantes, France. Nantes Université, CHU Nantes, Centre de Ressources et de Compétences des Maladies Hémorragiques Constitutionnelles, 44000, Nantes. CHU Angers, Centre de Traitement des Maladies Hémorragiques Constitutionnelles, 49000, Angers, France. CH Le Mans, Centre de Traitement des Maladies Hémorragiques Constitutionnelles, 72000, Le Mans, France. CHU Brest, Service Hématologie Clinique Hémostase, 29000, Brest, France. Nantes Université, CHU Nantes, Service Evaluation Economique et Développement des Produits de Santé, 44000, Nantes, France. Rennes Université, CHU Rennes, Centre de Ressources et de Compétences des Maladies Hémorragiques Constitutionnelles, 35000, Rennes, France. Inserm, EHESP, IRSET (Institut de recherche en santé, environnement et travail)-UMR_S 1085, F-35000, Rennes, France.

Publication : Clinical drug investigation. 2023

ABSTRACT: BACKGROUND: Von Willebrand disease (VWD) is the most common inherited bleeding disorder. However, studies of hospitalization 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 hospitalization. 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 hospitalized 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 hospitalization. These results could inform future studies of newly released recombinant VWF.