ASH WFH ISTH NHF VWD Guidelines – an international collaboration

Meeting a global need

The World Federation of Hemophilia (WFH) has joined a collaboration led by the American Society of Hematology (ASH), which also includes the International Society on Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), and the University of Kansas (KU) Medical Center to develop clinical practice guidelines on the diagnosis and management of von Willebrand Disease (VWD).

“Healthcare 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.”

VWD is the most common bleeding disorder, with approximately 1/1000 people experiencing symptomatic bleeding from VWD. This inherited condition, which effects women and men, results in the decreased production, absence, or abnormal function of the clotting protein, von Willebrand factor (VWF). 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 one person with VWD to another or in a single individual 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 people with mild symptoms do not receive a diagnosis right away and live for many years with untreated bleeding.

Why develop guidelines?

“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 the ISTH, NHF, and the WFH to convene diverse expert panels and expand the reach of the guidelines across the globe.”

Guidelines provide practical, evidence-based recommendations for healthcare providers, government authorities, payers, and patient organizations on how to diagnose and manage a condition. They can encourage people around the world to agree upon the best way to diagnose and manage the condition. If there is not enough evidence to back up a strong recommendation, guidelines can help to identify what research needs to be done. Clinical Practice Guidelines (also referred to as guidelines), are “statements that include recommendations intended to optimize patient care that are informed by a systematic review of evidence and an assessment of the benefits and harms of alternative care options.” (IOM, 2011)

Who will develop these VWD guidelines?

The goal of this international collaboration 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 international hematologists, people with VWD, and scientists with expertise in evidence synthesis and appraisal and guideline development methodology. The collaborating partners, ASH, the WFH, NHF, and the ISTH, all helped to identify the members of the panels. All panel members have undergone a thorough review for potential conflicts of interest. 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. The identities of the panelists will remain confidential until the guidelines are complete.

Who is sponsoring this project?

ASH is leading this project, with collaboration from the WFH, the ISTH and NHF. There are no corporate sponsors involved in this project and the development of these guidelines is wholly funded by the collaborating organizations. This is the first time that ASH, the ISTH, NHF, and the WFH have collaborated on clinical practice guideline development and dissemination.

“The ISTH is proud to partner with ASH, NHF, the WFH and the 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.”

How will these guidelines be developed?

There are international best practices for guideline development, including a systematic review of the available evidence and the evaluation of that evidence. ASH has contracted with KU Medical Center to do this review, so that the panels can develop guidelines based on the best information available.

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. The collaborating organizations will communicate opportunities for stakeholders to provide input on this prioritization.

Once the question formulation step is complete, the 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 healthcare providers implement the recommendations.

“This collaboration has the potential to be very impactful for our community,” commented 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 for becoming part of the guideline project speaks volumes to the potential of this initiative,” he added.

How can stakeholders contribute to these guidelines?

People living with VWD possess a unique expertise of the disease. The guideline development panels will be better able to make recommendations for diagnosis and management if they can learn from the experiences and expertise of as many stakeholders as possible. The collaborating organizations will soon be communicating opportunities for stakeholders to provide input, for example through an online survey. Keep an eye out for these communications and the chance to add your voice!

If you are a person with VWD and would like to be notified of opportunities to provide input for the development of these guidelines, please email Luisa Durante of the WFH at [email protected].

To read the ASH press release please click here.