Von Willebrand Disease | Diagnosis & Treatment

How is VWD diagnosed?

If your child is suspected of having von Willebrand Disease (VWD), they should be referred to a hematologist, a doctor with specialized training in treating blood disorders. Blood tests are needed to measure the levels and function of VWF. This include:

  • von Willebrand Factor antigen
  • von Willebrand Factor activity (commonly by Ristocetin cofactor activity)
  • Factor VIII activity

Additional diagnostic tests may be necessary depending on your child's individual situation. Once all necessary tests have resulted, our experts meet with you to review what they have learned about your child's results and to outline the best possible treatment options if needed.

What are the treatments for VWD?

The approach to VWD treatment depends on the VWD type and the individual’s severity and frequency of bleeding symptoms. Most often treatment is only needed prior to a procedure (such as surgery or tooth extraction) or for infrequent bleeding symptoms. In some cases, bleeding symptoms are more severe and more frequent treatment is necessary. The most commonly used treatments include:

  • Desmopressin (DDAVP): A synthetic hormone given by an injection into a vein or through a nasal spray called Stimate®. DDAVP causes an increased release of VWF from blood vessel cells. 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 prior to using it for treatment.
  • Von Willebrand Factor replacement therapies: Special concentrate of VWF (as well as factor VIII) are available for treatment of VWD. The VWF concentrates are administered by infusion (injections into a vein) and allow for replacement of VWF. This treatment is used if your child does not respond to DDAVP, needs therapy for an extended time, or has a severe injury.
  • Antifibrinolytic agents: Oral or IV medicines such as aminocaproic acid and tranexamic acid can be used to help make clots more stable. These medications may be particularly helpful for managing bloody noses (epistaxis), heavy menstrual bleeding, and after dental procedures for patients with bleeding disorders. An antifibrinolytic agent may be used alone or combined with DDAVP or VWF replacement therapy.

Your VWD treatment team will take many considerations into account to ensure the best treatment and follow-up plan for your child including:

  • your child's age, overall health and medical history
  • the severity of the disease
  • your child's tolerance for certain medications, procedures or therapies
  • how your child's doctors expect the disease to progress
  • your opinion and preferences

What is the long-term outlook for VWD?

Von Willebrand Disease is a lifelong condition with no cure. The long-term outlook for children with VWD is generally good and most have a normal lifespan. Throughout their lifetime, many patients require periodic treatment for bleeding symptoms or treatment before surgery or dental procedures. We generally recommend avoiding aspirin and ibuprofen (NSAIDS), as well as products containing them, since they may increase bleeding symptoms in children with bleeding disorders.