Real-world treatment patterns and outcomes in people with von Willebrand disease treated prophylactically with recombinant von Willebrand factor in the United States.

Background: People with von Willebrand disease (VWD) experience increased bleeding and decreased quality of life; those with a severe bleeding phenotype may benefit from prophylactic treatment. This retrospective chart review evaluated real-world effectiveness of prophylaxis with recombinant von Willebrand factor (rVWF) in all subtypes of VWD.

Research design and methods: People aged ≥12 years with a confirmed VWD diagnosis from US health care centers who received either routine or intermittent (for menorrhagia) prophylactic rVWF treatment were included. Eligibility criteria included availability of medical records ≥ 6 months pre- (baseline period) and post-rVWF initiation (rVWF treatment period). Annualized bleed rate (ABR), healthcare resource utilization (HCRU), and treatment patterns were the main outcomes of interest and were compared between both periods.

Results: Of 30 participants across 11 sites, 23 (76.7%) received routine rVWF prophylaxis for a mean duration of 2.9 years. Treatment is ongoing in most participants. ABR and total and bleed-related inpatient visits and number of surgeries decreased during the rVWF treatment period versus the baseline period.

Conclusions: Participants receiving routine rVWF prophylaxis in this study experienced reduced ABR and HCRU versus the baseline period, indicating that rVWF prophylaxis may result in improved outcomes in people with VWD across all subtypes.