SUZANNE O’CALLAGHAN
Suzanne O’Callaghan is HFA Policy Research and Education Manager.
Von Willebrand disease (VWD) figured prominently at the WFH 2026 World Congress, with experts discussing the advances to better understand and diagnose VWD, improve care, along with quality of life, and much excitement about new and emerging therapies for VWD.
Co-chairs ~ Saliou Diop, Senegal; David Lillicrap, Canada
VWD – 100 years in the making ~ David Lillicrap, Canada
Making the diagnosis ~ Ferdows Atiq, Netherlands
Current treatments ~ Michelle Lavin, Ireland
Future treatments ~ Caterina Casari, France
The von Willebrand disease session marked the 100th anniversary since Dr Erik von Willebrand published a report on ‘hereditary pseudohaemophilia’ in 1926 with the case of Hjördis Sundblom, a young girl from the Åland Islands near Finland, who displayed bleeding symptoms such as nose and ankle bleeds and later died from a bleeding episode when she began menstruating. He also analysed her family history and found many other members with a similar bleeding pattern, particularly females. This was later recognised as von Willebrand disease, the name of the condition acknowledging his work.
The session began with the comment that today the main disease burden of VWD remains in females who can menstruate.
Reflecting on the last 100 years, David Lillicrap explored the medical and scientific progress with VWD. He celebrated the achievements, including cloning the von Willebrand factor (VWF) gene and improved laboratory assays (tests) for VWF. He also outlined some unresolved issues:
Diagnosing VWD remains complex and Ferdows Atiq’s presentation outlined some of the current practices and challenges for the different types of VWD.
A person may be suspected of having VWD if they have a family history, bleeding that resembles VWD and abnormal laboratory results. Current practice is to then undertake further laboratory testing and evaluate bleeding with a standard bleeding assessment tool (BAT), such as the ISTH-BAT questionnaire. While these BATs are valuable when assessing bleeding severity, Atiq noted that they have some limitations – for example, BATs don’t give a greater weight to recurrent bleeding episodes in the same bleeding symptom category or account for changes in bleeding severity with age.
While some VWF laboratory tests are now more sensitive, some challenges remain with diagnosing VWD severity in particular. For example, he drew attention to:
Michelle Lavin, Chair of the WFH von Willebrand Disease and Rare Bleeding Disorders Committee, discussed current VWD therapies. She pointed out that diagnosis of VWD lags behind haemophilia, but that bleeds can have a large impact on quality of life.
Current therapies may include:
These therapies have their limitations. Some therapies are only appropriate for some people – for example, desmopressin is not suitable for people with type 3 VWD and most with type 2. Treatment failure is common in heavy menstrual bleeding and multiple treatment options may need to be combined, along with iron supplementation and folic acid. For those who are on regular prophylaxis, Lavin described the burden of very frequent infusions. She commented that unlike haemophilia, there was a lack of a peer culture in VWD supporting each other to have prophylaxis and some find it too hard to take on a continual basis.
Until recently development of therapies for VWD has been slow, but Caterina Casari presented a dazzling array of new VWD therapies in development. Some target different VWD types and include females in their clinical trials. It is hoped that these developments might be able to address some of the issues identified in this session.
Some of these new therapies under investigation were originally developed for other blood clotting disorders and are now being tested for people with VWD, such as emicizumab (Hemlibra®), which has been very prominent in treatment for haemophilia A.
Others are being developed specifically for VWD. They are in varying stages of development – for example, some are in very early stages, while VGA039 has commenced more advanced phase 3 studies and Casari commented that clinicians were looking forward to the publication of peer-reviewed data analysing the results.
| Novel investigational therapies for VWD1 | |
| Molecules increasing VWF levels | KBV13A12; HMB-002; BT200 |
| Agents enhancing the procoagulant [blood clotting] potential | |
| FVIII [factor 8] and mimetics | BIVV001; emicizumab; Mim8; NXT007; Inno8 |
| Rebalancing agents | VGA039; marstacimab; concizumab; ETX-148; fitusiran |
| Platelet-inspired nanoparticles | SynthoPlates |
| Antifibrinolytics | SG-100 |
| Genetic approaches | siRNA; gene editing; gene therapy |
| Molecules preventing excessive VWF degradation | Mab508; 3H9/17C7 |
The session finished on a high note. Although questions and challenges remain, there are exciting new developments and an international commitment to improving awareness, diagnosis, treatment and care for people with VWD.
FURTHER READING
WFH has published a State-of-the-Art journal article covering many of the points made in this session. You can read the full article here:
Lassila R, Atiq F, Casari C, Lavin M. Advances in von Willebrand disease. Haemophilia 2025; Supplement – State-of-the-Art: WFH 2026 World Congress. https://doi.org/10.1111/hae.70192
Suzanne O’Callaghan was assisted by funding from WFH to attend the WFH 2026 World Congress.
Sign up for the latest news, events and our free National Haemophilia magazine