For people newly diagnosed with VWD, the experience can feel 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.
The session began with Jessica Graham of the New York State Bleeding Disorders Coalition—who also lives with VWD—providing an overview. 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—a classification based on how much von Willebrand factor (VWF) they 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 by showing how treatment for VWD has evolved—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 – essentially making platelets “stickier,” which helps speed clot formation. 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 a common example of this therapy.
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