Why progress in von Willebrand disease treatment finally feels real

For decades, people with hemophilia have benefited from remarkable advances in treatment. Meanwhile, many people living with von Willebrand disease (VWD) have continued relying on therapies developed more than 20 years ago. Too often, VWD itself has been misunderstood or minimized.

Even getting diagnosed can be complicated. Unlike hemophilia, von Willebrand factor levels don’t stay consistent. Many factors can affect the amount of von Willebrand factor present in the blood, including stress, illness, hormones, age, exercise, and even blood type. A person can test in the normal range one year and in the diagnostic range the next.

My own levels have fluctuated widely over time. I happened to be tested during a period when they were low enough to confirm the diagnosis. Many people aren’t so lucky. They spend years trying to explain bleeding symptoms while lab results appear inconclusive, leaving them feeling dismissed or unheard.

I’ve watched too many people struggle for answers, battle heavy bleeding with limited treatment options, or be told their symptoms are simply “normal.” Many still don’t have access to specialists who truly understand VWD.

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But something important is finally changing. Researchers and pharmaceutical companies are now investing in new therapies that could dramatically improve treatment options — not only for people with VWD, but potentially for those with other rare bleeding disorders as well.

A recent article published in Haemophilia, the World Federation of Hemophilia’s official journal, notes that, for the first time, multiple new VWD therapies are entering or approaching clinical trials, offering real optimism for the future of VWD care.

That progress is exciting. But it also raises an important question: How do we ensure these advances reach the people who need them?

Lessons I’ve learned along the way

Don’t assume today’s treatment limitations are permanent. Many of us have been told, “This is just how it is.” But medical progress often moves slowly — until suddenly it doesn’t. Staying informed helps us advocate for better care as new options emerge.

Diagnosis still matters, even when testing is confusing. If bleeding symptoms affect your life, keep pushing for evaluation at a bleeding disorders center. Testing sometimes needs to be repeated, and expert interpretation makes a real difference.

Advocate for access, not just innovation. New therapies won’t help if insurance barriers or geography keep them out of reach. Patient voices matter in ensuring treatment advances are actually accessible.

Remember that bleeding disorders aren’t one-size-fits-all. VWD varies widely. Treatment decisions should reflect real-life bleeding experiences — not just lab numbers.

Hope matters. For many families, VWD has long felt like an afterthought compared with hemophilia care. Seeing real innovation finally happening sends a powerful message: This community matters, too.

We’re not at the finish line yet. Many patients still struggle to receive proper diagnosis and care. But for the first time in a long while, it feels like momentum is building.

And after generations of being told to simply deal with it, that hope feels long overdue.

Note: Hemophilia News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always consult your physician or another qualified healthcare provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Hemophilia News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to hemophilia.