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 to develop clinical practice guidelines on the diagnosis and management of von Willebrand disease (VWD).

VWD is the most common bleeding disorder, affecting approximately 1% of the world’s population. This inherited condition results in the decreased production, absence, or abnormal function of the clotting protein von Willebrand factor. VWD can cause unusual bleeding from small wounds or minor procedures, frequent nosebleeds, bruising, bleeding in joints, and heavy menstrual periods. Symptoms may vary from patient to patient or in a single patient over the course of his or her life. Primary care providers, pediatricians, obstetricians, and gynecologists who observe unusual bleeding often refer these patients to a hematologist for further testing and management. Many patients with mild symptoms do not receive a diagnosis right away and live for many years with untreated bleeding.

“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. “These guidelines will provide clinicians and patients with clear recommendations for the timely diagnosis of VWD and appropriate management of symptoms. ASH is pleased to collaborate with ISTH, NHF, and WFH to convene diverse expert panels and expand the reach of the guidelines across the globe.”

The goal of this effort is to create and maintain state-of-the-art guidelines on VWD. Two expert panels will produce recommendations for diagnosis and management. The panels are made up of more than 20 individuals, including US-based and international hematologists, patients with VWD, and scientists with expertise in evidence synthesis and appraisal and guideline development methodology.

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. In formulating questions, the panel will also place high importance on areas in which the development of guidelines could improve care and health outcomes important to patients and their caregivers.

Once the question formulation step is complete, KU Medical Center will conduct a systematic review of available evidence. Researchers with specialized training will identify, summarize, and grade the strength of the evidence. The panels will then use the evidence to develop recommendations for clinical practice. A public comment period will follow, with publication of the guidelines anticipated in 2020. Tools and educational resources will be released to help hematologists and other health-care providers implement the recommendations.

The majority of individuals participating in the guideline development have no direct financial interests in companies with products that will potentially be affected by the guidelines. Furthermore, development of these guidelines is wholly funded by the sponsoring organizations.

This is the first time that ASH, ISTH, NHF, and WFH have collaborated on clinical practice guideline development and dissemination.

“The ISTH is proud to partner with ASH, NHF, WFH, and KU Medical Center to develop important guidelines for the treatment of VWD as we address this global problem of diagnosis,” said Ingrid Pabinger, ISTH Chairman of Council. “The tools and resources that will be developed through this partnership will greatly help the hematology community and all related specialists and primary care physicians to improve patients’ level of care and health outcomes.”

“This collaboration has the potential to be very impactful for our community,” added Val Bias, CEO of NHF. “This collective effort will bring much-needed information to medical professionals and play an important role in improving outcomes that are vital to the health of our families. The enthusiasm from so many stakeholders to becoming part of the guideline project speaks volumes to the potential of this initiative.”

“Health-care professionals and our patient community have long identified a need for this kind of clinical guidance, and the WFH is very pleased to contribute to its creation,” said WFH President Alain Weill. “The diagnosis and treatment of VWD is a priority in our community and these guidelines should prove a valuable tool to physicians and hematologists working to improve the quality of life for those living with VWD as well as to the many ongoing essential VWD outreach and advocacy initiatives.”

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.