Clinical Practice Guidelines Being Developed for Rare Bleeding Disorder


Multiple hematology organizations have come together to develop state-of-the-art guidelines for the diagnosis and management of von Willebrand Disease.

A bleeding disorder that affects about 1% of the world population, von Willebrand Disease (VWD) often goes undiagnosed as symptoms of the disease range from mild to severe. As such, The American Society of Hematology (ASH) will collaborate with the International Society on Thrombosis and Haemostasis (ISTH), National Hemophilia Foundation (NHF), World Federation of Hemophilia (WFH), and the University of Kansas Medical Center will come together to develop clinical practice guidelines on the diagnosis and management of VWD to aid practitioners to improve the quality of life for those who are affected.

VWD is an inherited condition that affects an individual’s blood clotting ability. Specifically, the disease, “results in the decreased production, absence, or abnormal function of the clotting protein von Willebrand factor,” according to a statement on the collaboration from ASH. Individuals with the disease may experience symptoms that range from mild (bruising, prolonged nosebleeds) to severe (internal bleeding) depending on the type of the disease. The range of symptoms means that many patients will live for years with the disease without ever receiving a diagnosis.

“The complex clinical presentation of VWD is a challenge for hematologists and referring physicians. Many patients remain undiagnosed until they experience a severe bleeding episode that could otherwise have been prevented,” said ASH President Alexis A. Thompson, MD, MPH, of Ann & Robert H. Lurie Children’s Hospital of Chicago shared in the statement. She added that it is the hope of the collaborators that these guidelines will help clinicians to detect the disease early by providing clear recommendations on its diagnosis and the management of symptoms.

More than 20 internationally and US-based individuals from practitioners to patients to scientists comprise the 2 expert panels that will develop the guidelines. According to the statement, “panel members will identify and prioritize guideline questions based on the frequency with which a question arises in clinical practice, the degree to which there is variation in clinical practice, the extent to which the question has already been addressed by high-quality guidelines, and implications for resource utilization.” Those areas that will have the most impact on health outcomes and improved care will be the highest priority.

In an exclusive interview with Rare Disease Report®, Veronica Flood, MD, of Children's Hospital of Wisconsin, emphasized the signifiance of the collaborative forces. "Each of the areas [ of the guideline] has its own expertise and patient population that is coming together [through the 4 participating groups]," she said. "We have academic hematologists, community hematologists, methodology experts, content experts from fields related to these conditions, such as pediatricians gynecologists since women experience heavy menstrual bleeding and postpartum bleeding. Each of our panels will also have a number of patient representatives to weigh in and make these guidelines represent their guidelines as well."

The development of these guidelines is one part of a larger guideline development initiative by ASH that aims to develop evidence-based guidelines on a range of hematologic conditions. A public comment period is slated for 2019 and the final guidelines are expected to be published in 2020.

"I'm not sure there is a great standard of care [for Von Willebrand disease]," added Dr Flood. "There are definitely places that have closer to a standard of care than others. There was a previous guideline through the NHLBI, so there are some standards out there, but the goal is this [collaboration] will establish much better standards."

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