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Von Willebrand Disease

  • Von Willebrand disease (vWD) is an inherited bleeding disorder like hemophilia. It is caused by a defect or deficiency of a blood-clotting protein, called the von Willebrand factor. The von Willebrand factor is essential for the beginning stages of clotting and acts like glue to help the platelets stick together and form a blood clot.

    • Von Willebrand disease is the most common inherited bleeding disorder in the United States, believed to affect 1 to 2 percent of the population.
    • Von Willebrand disease is often compared to hemophilia. However, it is a different disease and is usually milder than hemophilia.
    • It affects both boys and girls equally, while hemophilia mainly affects boys. A parent with vWD has a 50 percent chance of passing the gene to his or her child.
    • Von Willebrand disease is a lifelong condition. With the available treatments, we can prevent excessive bleeding and help people lead normal, healthy lives.

    How Boston Children's Hospital approaches von Willebrand disease

    Your child will receive treatment for vWD at the Boston Hemophilia Center, the largest hemophilia program in New England.

    • The Boston Hemophilia Center is a joint program between Boston Children's Hospital and Brigham and Women's Hospital. Pediatric patients are seen here at BCH.
    • Our comprehensive care model ensures that every aspect of your child’s health is monitored by experienced pediatric caregivers.
    • Our goal is to enable children with hemophilia and their families to manage the illness as independently as possible, and therefore to lead more normal, healthy lives.

    Reviewed by Kapil Saxena, MD, and Ellis Neufeld, MD, PhD
    © Boston Children’s Hospital 2013

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  • What causes von Willebrand disease? 

    Von Willebrand disease (vWD) is caused by low levels of a blood-clotting factor called von Willebrand factor, and it helps blood to clot by acting as a kind of glue for platelets.

    The von Willebrand factor is one of a dozen substances in the body that contribute to the coagulation of blood. Factors are often designated by Roman numerals.

    What are the different types of von Willebrand disease?

    There are three main types of vWD:


    • Type 1—The most common and mildest form of vWD. Levels of von Willebrand factor are lower than normal, and the levels of factor VIII also may be reduced.   
    • Type 2—In type 2, the von Willebrand factor does not function the way it is supposed to. Type 2 vWD is divided into subtypes 2A, 2B, 2M and 2N. Each subtype is caused by different gene mutations and treated differently. This is why diagnosing the exact subtype is essential.
    • Type 3—The rarest and most severe type. People with type 3 vWD have virtually no von Willebrand factor and low levels of factor VIII, which means they are likely to experience serious bleeding.

    At the time of diagnosis, your child’s doctor will tell you which type she has and will outline the best options for vWD treatment.

    Risk factors for vWD

    The only risk factor for von Willebrand disease is a family history of this type of disorder. The gene responsible for the low levels of von Willebrand factor is inherited (passed down) from a parent. A parent with the gene for von Willebrand disease has a 50 percent chance of passing the gene on to his or her children.

    What are the symptoms of von Willebrand disease?

    Symptoms can vary a great deal depending on the type and severity of the disease. Many children with mild cases of von Willebrand disease have no visible symptoms. Others can experience symptoms such as:

    • frequent large bruises from minor bumps or injuries
    • frequent or prolonged nosebleeds
    • heavy or prolonged menstrual bleeding in girls
    • unusually prolonged or heavy bleeding from the gums during teeth-cleaning or other dental procedures
    • unusually heavy bleeding after surgery
    • blood in the urine or stools
    • excessive bleeding from minor cuts

    When to see a doctor

    You should call your doctor if you notice any of the above symptoms in your child.

  • How is von Willebrand disease diagnosed?

    If your child’s doctor suspects von Willebrand disease (vWD), she will ask about any family history of bleeding or bleeding disorders and will conduct a physical exam for signs of bleeding, liver disease or anemia.

    Your doctor will recommend certain tests to confirm the diagnosis. These tests are ordered by a hematologist (a doctor with specialized training in treating blood disorders). They are meant to measure the levels of von Willebrand factor in the blood, as well as the amount of other important factors and how well the body’s clotting mechanisms are working. These tests may include:

    • von Willebrand factor antigen
    • von Willebrand Ristocetin cofactor activity
    • factor VIII activity
    • von Willebrand multimers

    Your doctor may order these tests more than once to confirm the diagnosis and determine the proper course of treatment for vWD.

  • How is von Willebrand disease treated?

    Children with von Willebrand disease are treated through the Boston Hemophilia Center, the largest hemophilia treatment center in New England. This federally funded center provides comprehensive medical evaluation and treatment, ongoing medical management, counseling and support, and access to new treatment approaches through clinical research.

    Treatment of von Willebrand disease (vWD) may vary, depending on the type of vWD and the severity of the disease. If your child has mild vWD with no noticeable symptoms, treatment may only be needed intermittently, for surgery, tooth extraction or trauma.

    The most commonly used treatments for von Willebrand disease  include:

    • Desmopressin (DDAVP)—This is a synthetic hormone that is given by an injection into a vein or through a nasal spray called Stimate®. DDAVP controls bleeding by releasing more von Willebrand factor from storage sites in the body into your bloodstream. It is usually effective for most patients with type 1 and some with type 2 vWD. Your physician will usually perform a DDAVP challenge on your child to evaluate the response to DDAVP.
    • Von Willebrand factor replacement therapies—This involves infusion of a concentrate of von Willebrand factor into a vein. This is usually recommended if your child does not respond to DDAVP, needs therapy for an extended time or has type 2 or type 3 vWD.
    • Birth control pills—These pills can help control heavy menstrual bleeding by boosting the amount of von Willebrand factor and factor VIII (another clotting factor) in the bloodstream.
    • Antifibrinolytic drugs—These medications, such as aminocaproic acid (Amicar®) or tranexamic acid help prevent the breakdown of blood clots. They may be used alone or together with DDAVP and von Willebrand factor replacement therapy.

    If your child has vWD, anyone who takes part in her care—teacher, school nurse, daycare provider or coach—should be made aware of her condition.

  • The Boston Hemophilia Center is dedicated to advancing understanding and treatment of bleeding disorders like von Willebrand disease.

    • We currently offer several hematology clinical research studies to children with von Willebrand disease and other bleeding disorders.
    • Our scientists currently are conducting research that we hope will help to improve the lifestyle of our patients, including trials of new, longer-acting replacement factors aimed at reducing the frequency of required treatments.
    • In addition, we are investigating the possible use of gene therapy to treat von Willebrand disease, hemophilia and other bleeding disorders.

    Learn more about our hematology research.

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