Diagnostic and Treatment Availability of von Willebrand Disease Within Europe: Insights From a European Association for Hemophilia and Allied Disorders (EAHAD) Survey
Omid Seidizadeh, Ana Boban, Susie Shapiro, Riitta Lassila, Sophie Susen, Hamideh Yadegari, Laura Quintas Lorenzo, James S. O'Donnell, Giancarlo Castaman, Jeroen Eikenboom,ABSTRACT
Background
Von Willebrand disease (VWD) is the most common inherited bleeding disorder, yet its diagnosis and management remain heterogeneous across Europe due to variable access to diagnosis and care.
Aim
To characterize diagnostic and treatment practices for VWD across European hemophilia centers.
Methods
The European Association for Haemophilia and Allied Disorders (EAHAD) VWD Working Group conducted a cross‐sectional online survey among affiliated centers to evaluate center characteristics, use of bleeding assessment tools (BATs), availability of laboratory assays, genetic testing practices, diagnostic algorithms, and access to care and monitoring of treatments.
Results
Sixty‐two centers (27 countries) participated, being mostly comprehensive hemophilia care centers (77%). BATs were used by 85% of centers. Variability of VWF levels (57%) and limited access to genetic testing (29%) were the most frequently reported diagnostic challenges. VWF:Ag and FVIII:C assays were widely available (98%), whereas multimer analysis (39%), VWF:FVIIIB (31%), and VWFpp testing (13%) were less accessible. Genetic testing was performed routinely or selectively by 89%, most commonly using targeted next‐generation sequencing panels (65%). A VWF activity‐to‐antigen ratio threshold of 0.7 was used by 65% of centers to distinguish type 1 from type 2 VWD. While plasma‐derived VWF/FVIII concentrates were universally available, access to recombinant VWF (rVWF) was limited, with approximately one‐third of centers reporting no access. Prophylaxis was used predominantly for type 3 (78%) and cases with severe bleeding phenotypes regardless of subtype (63%).
Conclusions
Disparities in VWD diagnosis and care persist across Europe, highlighting the need for harmonized pathways and equitable access to specialized testing and therapies.