ボニコグアルファ対血漿由来フォン・ウィルブランド因子（入院期間中）：観察的後ろ向き多施設共同研究の結果

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, p = 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.