For people who have just discovered that they have VWD, the diagnosis can seem overwhelming. The Thursday pre-conference session, “VWD: Your Masterclass in VWD Insights and Community,” sponsored by CSL Behring, was designed to give attendees a firm understanding of the bleeding disorder, current treatments, and what future therapies are being studied.
Attendees took seats at round tables, and at each place was a little silicone finger puppet. The puppet, explained Michael GrandPre, represented your “inner doubt monsters.” He encouraged attendees to keep the puppet to help conquer inner doubts.
Kim Walsh, a lived experience expert, shared her story, including her diagnosis of VWD at age 35 and the challenges she faced before finally receiving proper treatment.
Jessica Graham of the New York State Bleeding Disorders Coalition – who herself has VWD – next took the stage to give an overview of VWD. VWD is the most common inherited bleeding disorder, affecting up to 1% of the US population, and although it is inherited equally by men and women, it often disproportionately affects women through heavy periods and post-partum bleeding.
How a person experiences VWD depends on their type – which is a loose way of categorizing how much von Willebrand factor (VWF) they can produce. Von Willebrand factor is a protein that helps blood cells stick together at the site of an injury. It also carries factor VIII in the bloodstream, protecting it until that protein is needed in the clotting cascade. Type 1 is considered mild, type 2 is intermediate, and type 3 is the most severe.
Veronica Flood, MD, of the Medical College of Wisconsin, educated the attendees on the history of treatment for VWD and where treatment currently stands today.
She began with showing how treatment of VWD has evolved over time – going back to the 1900s when things such as snake venom might be “prescribed” to treat bleeds. But by the mid- to late-20th century, the advancements gained speed, from cryoprecipitate to VWF concentrate, eventually leading to recombinant VWF in the 2010s.
There are four methods to treat VWD, and which one a person uses depends on what type of bleeds they experience and how severe they are.
One method is replacement or mimic therapies – which gives the body more von Willebrand factor (or something that acts like it). Plasma-derived concentrates like Humate-P and Wilate, or recombinant VWF like Vonvendi are examples of this. There are medicines that prime platelets and enhance clot formation – or making platelets “stickier,” which can speed up clotting. Desmopressin is an example of this strategy. Reducing fibrinolysis, or stopping the body from breaking down clots, is typically used for mucosal bleeds, such as nose and mouth bleeds, and heavy menstrual bleeding. Tranexamic acid is an example of this therapy.
Hormonal birth control, often prescribed to treat the heavy menstrual bleeding that is a symptom of VWD, doesn’t actually change how your blood clots. It just reduces how much blood needs to be clotted by making the uterine lining thinner.
Robert Sidonio, MD, MSc, from Emory University, gave an overview of what the future might hold for VWD care. There are new and evolving treatment options for managing VWD.
He began by pointing out that even though there are clinical guidelines for the treatment of VWD issued in 2021 that recommended prophylaxis to prevent bleeding for patients who experience bleeds, most people in the US who have severe VWD do not treat prophylactically. This means that most people are treating bleeds after they happen – not taking medicine to prevent bleeding in the first place.
Compared with the treatment of hemophilia A, for example, 45-85% of people diagnosed as severe receive prophylaxis, compared to 10% of those with severe VWD.
Currently, there are four FDA-approved medications that contain VWF that can be used to treat VWD, but only two are indicated for prophylaxis, Wilate and Vonvendi. There are currently several clinical trials for Vonvendi and Wilate to determine the long-term safety and efficacy of these therapies.
Vagus nerve stimulation is a novel approach that is showing early promise. Neurostimulation is used to “prime platelets in the spleen,” improving symptoms of heavy periods.
Because of the role that VWF factor plays in transporting FVIII, some studies are currently looking to see if new therapies developed to treat hemophilia A would work on VWD. Roche is currently enrolling people with VWD in a clinical trial to see if emicizumab, which mimics FVIII, is effective for treating people with VWD.
Another angle being studied is increased thrombin generation. VGA039 is a subcutaneous therapy that boosts thrombin generation to enhance clot formation. This therapy has the potential to treat different VWD types. A clinical trial to study this is currently accepting enrollees.
Dr. Sidonio concluded by stating that further studies are being conducted to investigate the role of personalized prophylaxis, based on mathematical models, to find dosing schedules that work for most people, and can be fine-tuned for individuals.
