Von Willebrand Disease

What is von Willebrand disease?

Von Willebrand Disease (VWD) is an inherited bleeding disorder caused by an abnormal blood-clotting protein called von Willebrand Factor (VWF). The VWF is important for the body to create the first steps of a blood clot to stop bleeding with injury and surgery. It’s particularly important for helping to making needed blood clots when one has a bloody nose, mouth bleeding or a monthly period. VWF acts like glue to help the platelets stick together and form a blood clot. VWF is one of several clotting proteins in the body that must work together to prevent abnormal bleeding.

VWD is one of several bleeding disorders. Others include hemophilia, platelet disorders, and other factor deficiencies. It is important to know that different bleeding disorders have different risks of bleeding and different patterns or common locations of bleeding. VWD is the most common inherited bleeding disorder and affects about 1 in every 1000 individuals. Males and females have an equal chance of inheriting VWD; however, due to the bleeding challenge of monthly menses (periods), VWD is more frequently diagnosed in females. Some individuals can have very mild decreases in the VWF without any (or very minimal) bleeding symptoms; these individuals are not considered to have VWD.

Parents with VWD has a 50% chance of passing the abnormal VWF gene to their child. VWD is a lifelong condition that currently does not have a cure but can be successfully managed with several types of treatments available to manage and prevent bleeding.

Are there different types of VWD?

Yes, broadly there are three types of von Willebrand disease: Types 1, 2, and 3. There are also subtypes of type 1 and type 2. Type 1 VWD is most common.

  • Type 1 VWD: VWF are lower than normal (<30%). This is measured my both amount (antigen level) of VWF as well as by function (activity level) of VWF.
  • Type 2 VWD: VWF is often in the low or low-normal range but VWF does not function normally. There are several subtypes noted by different letters (2A, 2B, 2M, and 2N). Each of these subtypes are defined by the specific function of VWF that is not working properly, for example VWF’s ability to interact with platelets or with factor VIII (8). Specialty testing is needed to identify the subtype.
  • Type 3 VWD: VWF is absent. This is the rarest and most severe type of VWD. In these patients, bleeding symptoms are similar to those of moderate hemophilia.

How we care for VWD

We treat our patients who have von Willebrand Disease within the Boston Hemophilia Center, a federally-supported joint program between Boston Children’s Hospital and Brigham and Women's Physicians Organization. The Boston Hemophilia Center is the largest hemophilia treatment program in New England, and it provides treatment for a variety of bleeding disorders, including VWD.

Our team helps children and adults with bleeding disorders to manage their disease as independently as possible and supports the wellness of each individual. Living a healthy life and achieving your goals is possible. Our patients receive the benefit of access to cutting edge treatments pioneered by our researchers and the national network of hemophilia treatment centers (HTCs).

Our areas of research for VWD

For many children with rare or hard-to-treat conditions, clinical trials provide new treatment options. Participation in clinical trials has risks and benefits, but it is through clinical trials and clinical research studies that we are able to forward the field of VWD and other bleeding disorders.

Participation in a clinical trial is always voluntary. The doctor leading the clinical trial (study) at the site will review with you in detail the purpose of the study and what is required of you/your child during the course of the study before you agree to participate. Search our clinical trials or contact us if you’re not sure which clinical trials might be right for your child. We can help you navigate your options.