PB0845 - Healthcare Resource Utilization in Patients with von Willebrand Disease Receiving Recombinant von Willebrand Factor On Demand and in Surgical Settings: Results From a European Chart Review

Background: Limited real-world data are available describing healthcare resource utilization (HRU) in patients with von Willebrand disease (VWD) receiving recombinant von Willebrand factor (rVWF; vonicog alfa).

Aims: To assess HRU in patients receiving rVWF on demand (OD) or in surgical settings.

Methods: This observational, retrospective study involved adults (age: ≥18 years) with congenital VWD or symptomatic low VWF levels (30–50%, VWF antigen test) from 6 European countries. Eligible patients must have received rVWF OD to treat ≥1 spontaneous/traumatic bleed and/or to prevent/treat surgery-related bleeding (date of first rVWF dose [index]: 01/01/2019–31/10/2020). Exclusion criteria: other bleeding disorders or factor deficiency (including acquired von Willebrand syndrome), VWF neutralizing antibodies, and intermittent/long-term plasma-derived VWF or rVWF prophylaxis during the 12 months before index or during the post-index period (3–12 months; from index until death, loss to follow-up, or study end). VWD-related HRU data (including hospitalization rates, length of hospital stay, outpatient attendances, and laboratory tests) were collected from medical records. Patient informed consent (where applicable) and ethics approval was obtained. Herein, we report HRU at index for patients who received rVWF.

Results: 82 patients from 13 sites had HRU data recorded; 53 were female, mean ± SD age at index was 47.7 ± 16.5 years, and most patients had type 1 (n=31) or type 2 (n=42) VWD. Hospitalization and outpatient attendance were required by 62% and 39% of patients receiving rVWF to treat spontaneous/traumatic bleeds, and by 75% and 26% of those receiving rVWF to prevent/treat surgical bleeds, respectively (Table 1).

Conclusion(s): To our knowledge, these data provide the first insights into real world HRU for patients with VWD receiving rVWF to treat spontaneous/traumatic bleeds or to prevent/treat surgical bleeds. These data can be used to support further research to understand differences in HRU between treatment cohorts or centers, and give stakeholders HRU estimates for future decision making.