New clinical practice guidelines for the diagnosis and management of von Willebrand disease

The American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) have developed joint clinical practice guidelines on diagnosis and the management of von Willebrand’s disease (VWD), the world’s most common inherited bleeding disorder. The guidelines were published today in Blood advances.

Von Willebrand disease affects about 1% of the world’s population and is the most common bleeding disorder. Although von Willebrand disease also occurs in men and women, women are more likely to notice symptoms due to heavy or abnormal bleeding during their period and after childbirth.

This inherited disease causes a decrease in the production, absence or abnormal function of von Willebrand factor, a coagulation protein.

Von Willebrand disease can cause unusual bleeding from small wounds or minor procedures, frequent nosebleeds, bruising, joint bleeding and heavy menstruation and postpartum bleeding in women. Symptoms may vary from patient to patient or in a single patient over the course of their life.

Primary care providers, pediatricians, obstetricians, and gynecologists who observe unusual bleeding often refer their patients to a hematologist for further testing and management. Many people with mild symptoms don’t get diagnosed right away and live for many years with untreated bleeding or don’t realize they have von Willebrand disease until they have one. severe bleeding that could have been avoided.

“Although von Willebrand disease is a common bleeding disorder, it is also complex, presenting challenges in the rapid diagnosis and appropriate management of bleeding for patients,” said ASH 2021 President Martin S. Tallman, MD, of Memorial Sloan Kettering Cancer Center.

“Because diagnosis is not straightforward and symptoms vary in severity, there is a need for reliable guidelines to help improve the quality of patient care. There are no better partners for ASH in this effort than ISTH, NHF and WFH, whose collective expertise in von Willebrand disease from the perspectives of scientists and patients around the world. reinforced this effort. “

Notably, the guidelines make key statements about laboratory test cutoffs to classify von Willebrand disease as more inclusive of patients who experience bleeding, but whose blood tests do not meet currently accepted diagnostic thresholds.

In addition, the guidelines recommend for the first time that patients with von Willebrand disease who suffer from frequent and severe bleeding that diminishes quality of life receive routine prophylaxis for von Willebrand disease – an injectable concentrate. clotting protein – several times a week. Together, the diagnostic and management guidelines include 19 combined recommendations.

The guidelines were developed by two expert panels comprised of 32 people, including US and international hematologists, people with von Willebrand disease, and scientists with expertise in synthesizing and evaluating evidence and the methodology for developing guidelines.

The clinical questions were developed by the panels and prioritized, and an international survey was conducted to identify the most important clinical questions. A systematic review of the available evidence was conducted by the University of Kansas Medical Center and the committee referred to this evidence in making recommendations.

This process has highlighted the lack of solid evidence on which to base recommendations, and the report therefore calls for more research.

“These guidelines are an excellent example of collaboration between several companies ensuring that the guidelines developed represent the most up-to-date and relevant advice on the diagnosis and treatment of this common but often misunderstood bleeding disorder,” said Claire McLintock, MD, past president of ISTH.

“Working with the amazing team that developed the guidelines for the diagnosis of von Willebrand disease has been extremely rewarding and inspiring.

People with von Willebrand disease made up about a quarter of the makeup of each recommendation group. As full voting members, they brought their expertise to all aspects of the rigorous guideline development process, from prioritizing clinical questions, to assessing the available evidence, to making taking into account factors such as stakeholder values ​​and preferences, equity and feasibility. .

Their involvement, actively supported by the panel chairs and collaborating organizations, has been essential to ensure the development of recommendations with the greatest relevance and in which the community will have great confidence. Collaboration with the WFH and NHF was essential to engage the wider community of patients with von Willebrand disease.

Through their extensive functional networks established with healthcare professionals in comprehensive bleeding disorder care centers and patient organizations in the United States and around the world, the four collaborating organizations, ASH, ISTH, NHF and WFH will all play an important role in the creation and dissemination of resources based on the guidelines, and their implementation in individual care and broader advocacy efforts.

“These guidelines are an extremely important step in our quest to address the challenges individuals face in obtaining an accurate, timely diagnosis and appropriate treatment. The challenge ahead will be for us to educate both people with von Willebrand disease and healthcare professionals on the guidelines. recommendations, ”said Leonard Valentino, MD, president and CEO of NHF.

We were excited to work with ASH, ISTH and WFH to create them and present them to the community. In 2017, we brought together members of the global VWD community for the first international WFH symposium on von Willebrand disease. Delegates from countries around the world have told us that the number one tool they need to provide comprehensive care for people with von Willebrand disease is clinical practice guidelines. “

Cesar Garrido, President, World Hemophilia Federation

“The WFH is very proud to provide this guidance today, along with our partners ASH, ISTH and NHF, to the global community. In addition to providing advice to clinicians and people with von Willebrand disease, they are involved in making personalized care decisions. basis of important advocacy initiatives to improve access to diagnostic techniques and recommended treatment options in all countries. “

The publication of the guideline will be accompanied by educational tools and resources to help patients, hematologists and other healthcare providers understand and implement the recommendations.