Work

Outcomes and Healthcare Resource Utilisation in Adults With von Willebrand Disease Receiving On‐Demand Recombinant von Willebrand Factor in the United Kingdom

Year: 2025

Type: article

Abstract: OBJECTIVES: We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) treated on demand with recombinant von Willebrand factor (rVWF) in the United Kingdom. METHODS: Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first-time rVWF for the on-demand treatment of spontaneous/traumatic bleeds, or the prevention/treatment of surgical bleeds, between 1 October 2020 and 30 June 2022 at seven hospitals. Treatment, outcome and VWD-related HCRU data associated with the on-demand treatment of all recorded bleeds were collected at first (index) treatment, for 12 months pre-index and 3-12 months post-index. RESULTS: Twelve patients (91.7% female and White/Caucasian, mean age 34.2 years) were treated on demand with rVWF at index: bleed resolution was achieved for all bleeds with limited requirement for additional treatments, no treatment switches, and no complications reported. Physician-rated treatment satisfaction was 'excellent' (82.6%) or 'good' (17.4%) for all recorded on-demand rVWF-treated bleeds (n = 23). Patients receiving any on-demand treatment required an inpatient admission and ≥ 1 outpatient visit in 33.7% and 66.7% of cases, respectively. CONCLUSIONS: Results support the effectiveness and safety profile of on-demand rVWF treatment for spontaneous/traumatic bleeds in adults with VWD, adding to the growing body of evidence for rVWF.

Source: European Journal Of Haematology

DOI: 10.1111/ejh.70032