• von Willebrand Disease Overview

    Von Willebrand disease (vWD) is an inherited bleeding disorder caused by low levels of a protein called von Willebrand factor, which helps blood to clot by acting as glue for platelets. It is often compared to hemophilia, but is a different disease and usually milder than hemophilia. It is the most common inherited bleeding disorder in the United States. There are three main types of vWD:

    • Type 1 – the most common and mildest form in which there are lower than normal levels of von Willebrand factor and, possibly, reduced levels of factor VIII in the blood
    • Type 2 – in which von Willebrand factor does not function the way it is supposed to; includes subtypes 2A, 2B, 2M and 2N, which are caused by different gene mutations and treated differently
    • Type 3 – the rarest and most severe type in which patients have virtually no von Willebrand factor and low levels of factor VIII, which means they are likely to experience serious bleeding.

    von Willebrand Disease Treatment at Dana-Farber/Boston Children's

    Children and young adults with vWD are treated at Dana-Farber/Boston Children's Cancer and Blood Disorders Center through our Hemophilia Program. Continue reading to learn more about von Willebrand disease or visit the Hemophilia Program homepage to learn about our expertise and treatment options for this condition.

    Symptoms & Diagnosis

    Many children with mild cases of vWD 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 stool
    • Excessive bleeding from minor cuts

    If a doctor suspects a child of having  vWD, he will ask about any family history of bleeding or bleeding disorders. In addition to a physical exam to check for signs of bleeding, liver disease or anemia, tests to measure the level 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, may be done. These include:

    • von Willebrand factor antigen test
    • von Willebrand ristocetin cofactor activity test
    • factor VIII activity test
    • von Willebrand multimers

    A doctor may order these tests more than once to confirm the diagnosis. After all tests are completed, doctors will be able to outline the best treatment options.

    Treatment & Care Options

    Treatment of vWD varies, depending on the type and severity of the disease. If a child has mild vWD with no noticeable symptoms, treatment may only be needed for surgery, tooth extraction or trauma. The most common treatments used for vWD include:

    • Desmopressin or DDAVP – a synthetic hormone that controls bleeding by releasing more von Willebrand factor from storage sites in the body into the blood stream; effective for most patients with type 1 and some with type 2 vWD
    • vWD replacement therapies – an infusion of a concentrate of von Willebrand factor into a vein; recommended for children who do not respond to DDAVP, need therapy for extended time, or have type 2 or type 3 vWD
    • Birth control pills – to control heavy menstrual bleeding by boosting levels of von Willebrand factor and factor VIII in the blood stream
    • Antifibrinolytic drugs – to help prevent the breakdown of blood clots; may be used alone or in combination with DDAVP and replacement therapy

    Long-term Outlook

    The long-term outlook for children with vWD is generally good and most have a normal lifespan. Throughout their lifetime, many patients require periodic acute treatment for active bleeding or before medical procedures. To reduce the risk of complications, patients should avoid pain relievers such as aspirin and ibuprofen and blood-thinning medications such as Plavix and Coumadin, and stay active.
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