Seminars in thrombosis and hemostasis · 2026
Vonicog Alfa versus Plasma-Derived von Willebrand Factor during Hospitalization: Results of an Observational Retrospective Multicenter Study.
СтатьяМногоцентровое исследованиеНаблюдательное исследованиеНазвание и аннотация: Перевод в очереди
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Horvais Valérie, Ternisien Catherine, Denis-Le-Sève Julien, Beurrier Philippe, Fouassier Marc, Babuty Antoine, Drillaud Nicolas, Pan-Petesch Brigitte, Rose Johann, Cussac Vincent, Bayart Sophie, Guillet Benoît, Trossaërt Marc
Аннотация
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's 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.
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's 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.