Key result
Patients with von Willebrand disease who were eligible for but not receiving prophylaxis experienced significantly more treated bleeds (mean 5.5 vs 1.5) and hospitalizations due to bleeding compared to those receiving prophylaxis.
Why the study?
Recent international guidelines conditionally recommend von Willebrand factor prophylaxis for patients with von Willebrand disease and severe or frequent bleeds, prompting assessment of disease burden in potentially eligible patients.
Does von Willebrand factor prophylaxis reduce disease burden in patients with congenital von Willebrand disease and severe/frequent bleeds?
Cross-Sectional (n=331)
Yes
Does von Willebrand factor prophylaxis reduce disease burden in patients with congenital von Willebrand disease and severe/frequent bleeds?
Absolute Event Rate: 5.5% vs 1.5%
p-value: p=<0.001
Patients with congenital VWD who are eligible for but not receiving VWF prophylaxis experience a significantly higher disease burden than those on prophylaxis, supporting guideline recommendations for its use.
Patients eligible but not on VWF prophylaxis had higher bleeding and joint burden; leaves open whether prophylaxis reduces disease burden in congenital VWD.
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.
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Du et al. (2022) conducted a cross-sectional in von Willebrand disease (n=331). No VWF prophylaxis (prophylaxis-eligible) vs. VWF prophylaxis was evaluated on Mean number of treated bleeds in the 12 months before the index date (p=<0.001). Patients with von Willebrand disease who were eligible for but not receiving prophylaxis experienced significantly more treated bleeds (mean 5.5 vs 1.5) and hospitalizations due to bleeding compared to those receiving prophylaxis.
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