VWD Medical Expertise Lacking
What is the state of medical knowledge in terms of how to treat VWD, and what's being done to broaden that knowledge?
ANDRA: Treatment knowledge appears to be limited to a very few experts in hemostasis. We train a lot of hematologists in this country, 90 percent of whom specialize in cancer treatment. So only 10 percent really practice something other than oncology, and among them, just 10 percent are experts in hemostasis. There aren't any other specialists coming in to fill the void, either. We rely on experts in hemostasis, both pediatric and adult hematologists, to inform other practitioners on how to handle our patients who suffer from bleeding problems.
ALAN: The NHF is part of a coalition with the U.S. Centers for Disease Control and Prevention, the American College of Obstetrics and Gynecology, and the American Society of Hematology. This consortium communicates new information that's available to physicians and other treaters. This consortium also has the ability to focus on the various entry points of potential VWD patients into the healthcare system. We hope that vigilance, along with Project Red Flag, will make the general public more aware of bleeding disorders and related issues.
PAUL: The education process doesn't end with, "Okay, I need to look for von Willebrand disease." It also entails, "When and how do I treat VWD if I diagnose it?"
ALAN: Right now, there's no well-validated test for von Willebrand disease, which usually requires multiple layers of testing to diagnose.
PAUL: Yes, and there doesn't seem to be a universal consensus or easy way to get the testing done. Some labs tend to do it better than others, and it depends on what you're looking for. This pertains both to testing and classifying to determine what type of VWD is present. Is it Type 1, Type 2, Type 3? And does that make a difference in terms of who you treat? It's a difficult condition to get consensus on. This is why I think that the guidelines being developed by the National Heart, Lung, and Blood Institute will really help increase understanding about diagnosis and proper treatment of von Willebrand disease.
You are referring to the guidelines on diagnosing and treating women with VWD. When are these guidelines expected to be ready, and what kind of impact will they make?
ANDRA: I expect the guidelines to be available in the next few months, but they won't contain any surprises. The NHLBI didn't suddenly decide that there was a magical screening or diagnostic test that's available, but there is consensus on the types of questions that ought to be asked of patients, what kind of tests ought to be ordered first line and second line, and which elements should be taken into consideration in making a diagnosis. In some respects, it will be a gold standard for future studies, but it's going to be a work in progress. Consideration ought to be made toward updating and revising the guidelines every three or four years or so. This will accommodate advances or changes in diagnostic methods or treatment. The NHF can be particularly helpful in distributing the guidelines to practitioners. Our partners in industry can also help disseminate the information. Historically, when we've tried to educate around von Willebrand disease, people get hung up on the controversies, and the main message gets missed. The guidelines will say that the data are weak here, or we don't have evidence for this, or this is opinion, or this is controversial. From there, we can move on so that we get the main message out.
ALAN: Project Red Flag has identified some best-practice models in disseminating information, such as the peer-education program, Woman-to-Woman. This program trains women to speak about their bleeding disorders and to serve as a resource connecting other women with similar symptoms to NHF's local chapters and hemophilia treatment centers across the country. The NHLBI guidelines should focus the medical and scientific communities on the serious issues, like the need for simple, accurate, reproducible diagnostic testing. In terms of medical practice, the best methodologies and best course of treatment have certain questions remaining. I hope these guidelines will focus the federal government on supporting more research into von Willebrand therapy. Better Treatment Approaches Needed
What challenges arise when caring for pregnant women with VWD?
ANDRA: As we beat the bushes for more patients, we need to do a better job with the patients we have. My career is about trying to prevent complications. We are just really ignorant about what adequate prophylaxis is.
What can we do to prevent complications?
ANDRA: I need more data. I need more experts to join me in the field. We need to get them trained. We need some obstetrician/gynecologists who have an interest in this, too. I also think that these guidelines aren't necessarily geared toward patients or consumers, although they will be the beneficiaries, and they aren't geared toward hemostasis experts, who can argue about how many platelets you can fit on the head of a pin. They are geared toward primary practitioners—pediatricians, OB/GYNs, family practitioners, nurse practitioners and people who are in the field. I hope that the guidelines and everything else we do to educate our colleagues will demystify the condition so that it's accessible, so that it's discussible, so they can identify the best laboratory to get the appropriate tests and know who would be the best person to help them.
What are the data gaps, in terms of being able to identify and treat patients?
ANDRA: Is a woman with von Willebrand disease more likely to miscarry? We just don't know. How quickly do von Willebrand factor levels that rise during pregnancy fall afterward? When are the true vulnerable periods? How long after childbirth should we use prophylaxis? I thought 12 days was best, but maybe it's more like three weeks. ZLB Behring will help us get the answers, and the medical advisory committee has given its blessing to developing a proposal that would be funded to study postpartum prophylaxis. This is the kind of research that needs to be done, not just things in a test tube.
ALAN: NHF clearly supports all of these efforts to expand our body of knowledge. As diagnosis and treatment of VWD improves, we will help educate healthcare providers and consumers, as well as the general public and those who make policy decisions in Washington.
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