Severe VWD: prophylaxis reduces bleeding

The clinical evidence in favour of prophylaxis for von Willebrand disease has so far been sparse. This is changing with the data from the WIL-31 study.

Von Willebrand disease: the most common inherited bleeding disorder

VWD is the most common inherited bleeding disorder with a prevalence of 0.6 % to 1.3 %. Haemostasis is impaired due to a deficiency or dysfunction of the von Willebrand factor (VWF). The severity of the haemorrhages varies greatly and ranges from mild to severe.

Long-term prophylaxis (well established for haemophilia) is also recommended by international guidelines for severe VWD^.2 The aim of prophylaxis is to minimise bleeding events, reduce joint damage and improve quality of life.

WIL-31-Studie: Prophylaxe mit FWF-FVIII-Präparat

The prospective, non-controlled, international phase 3 study WIL-31 investigated the efficacy and safety of prophylaxis in 33 adult and paediatric VWD patients (von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease, DOI: 10.1182/bloodadvances.2023011742). Treatment was with a VWF/FVIII concentrate. The results of a preceding study in which the participants received on-demand therapy with VWF/FVIII concentrate served as a comparison^.1

84% reduction in bleeding

The primary endpoint, defined as a >50% reduction in the mean annual total annualised bleeding rate (TABR) with 12 months of prophylaxis compared to the previous on-demand treatment, was achieved: the mean TABR during prophylaxis was 5.2, which corresponds to an 84.4% reduction. The reduction in bleeding was comparable across age, gender, and VWD types. The prophylaxis was well tolerated and did not lead to thrombotic events or deaths.

Prophylaxis prevents heavy menstrual bleeding

Although women are less likely to develop VWD than men, menstrual bleeding is a major problem when they are affected. 5 out of 7 women included in the WIL-31 study benefited from the prophylaxis. They did not have heavy menstrual bleeding during the prophylaxis.

Conclusion: Data provide good arguments in favour of wider use of VWF prophylaxis

• Prophylaxis with a VWF/FVIII concentrate reduced the bleeding rate in severe VWD compared to on-demand therapy.
• The WIL-31 study thus provides good arguments in favour of the wider use of VWF prophylaxis in VWD patients.

[Abbreviation: FVIII: Factor VIII]