Disease Burden in Patients with von Willebrand Disease Potentially Eligible for Prophylaxis: Post Hoc Analysis of a European Cross-Sectional Study.

Recent international guidelines conditionally recommend von Willebrand factor (VWF) prophylaxis for von Willebrand disease (VWD) patients with a history of severe/frequent bleeds. This post hoc analysis of the Cost of VWD Across Europe, a Socioeconomic Study (CVESS; conducted in 2018), assessed patient characteristics and disease burden in patients aged >1 year with congenital VWD not receiving but potentially eligible for prophylaxis based on severe/frequent bleeds, and those receiving prophylaxis in the previous 12 months. Data were collected using medical records and a patient questionnaire. Patients considered potentially prophylaxis-eligible (n = 102) experienced more bleeds than patients receiving prophylaxis (n = 229) and were more likely to be admitted to the hospital due to bleeding events in the prior 12 months. Quality of life and work productivity were similar between the two groups. Logistic regression analysis showed that the prophylaxis-eligible group was more likely to have poor joint function and moderate chronic pain than the prophylaxis group. This retrospective study suggests that 1/7 patients not receiving VWF prophylaxis had a higher disease burden than patients receiving prophylaxis and would potentially benefit from prophylaxis.