Von Willebrand Disease

What is Von Willebrand Disease?

Von Willebrand Disease (VWD) is an inherited disorder that you are born with. In normal blood, proteins, called clotting factors, work together to form a clot when there is bleeding. A person with VWD either doesn’t have enough of one of these clotting factors, or the clotting factor doesn’t work right. For someone with VWD, the missing or defective clotting factor is called von Willebrand factor (VWF). VWF has two jobs:

  • It acts like glue to help platelets stick together at the site of an injury to form a platelet plug.
  • It carries Factor VIII (another clotting factor) to the site of an injury so that a fibrin clot can be met.

VWD is the most common bleeding disorder, affecting approximately 1% of the population, or 3.2 million people in the United States.

VWD affects males and females equally. However, females are more likely to be diagnosed as they have obvious symptoms such as heavy menstrual bleeding. Many individuals, especially those with mild symptoms, may never be diagnosed or are diagnosed later in life.

Types of Von Willebrand Disease

Type 1 is the most common type and the mildest form.

  • There is a lower than normal VWF level.
  • Factor VIII (8) levels may also be low.

Type 2 has a normal amount of VWF, but it doesn’t work the way it is supposed to work. There are four subtypes.

  • Type 2A
    • The VWF is not the right size.
    • The VWF doesn’t glue the platelets together to form a platelet plug.
  • Type 2B
    • The VWF attaches to platelets when there isn’t an injury.
    • The body removes the used VWF leaving too little available when it is needed to form a platelet plug.
  • Type 2M
    • The VWF isn’t able to stick to platelets.
    • Without VWF attached to the platelets, a platelet plug can’t form.
  • Type 2N
    • The VWF does not attach to factor VIII (8) and bring it to the site of an injury.
    • The unattached factor VIII (8) is removed from the bloodstream.
    • A fibrin clot cannot form.

Type 3 is the rarest and most severe form of VWD.

  • There is little to no VWF.
  • Factor VIII (8) levels are low.
  • Neither a platelet plug nor a fibrin clot can be formed.
Treatment for Von Willebrand Disease

Although there is currently no cure for VWD, there are good treatments available to prevent and/or stop bleeding. You and your doctor (hematologist) will talk about a treatment plan and medication that is right for you.

DDAVP is a medication that triggers the body to release more VWF and also more factor VIII (8) in the blood.

  • It is used to treat Type 1 VWD.
  • DDAVP does not work the same for every person. Your doctor will give you a test dose at the clinic and test your blood several times on the same day to see if it works for you.
  • It can work well for heavy periods, dental procedures, or minor procedures but for more complex procedures some patients may need clotting factor replacement. 
  • Can be given into a vein, as a shot, or as a nasal spray.
  • DDAVP is not recommended for young children or the elderly.

Clotting Factor Replacements are medications that replace the VWF and/or factor VIII (8) so that the blood can clot correctly.

  • Clotting factor is infused (given directly into a vein or in a port).
  • Prophylactically – infusing factor on a regular schedule to prevent bleeding from happening.
  • On-Demand – infusing factor during a bleed to make it stop.
  • Some patients have both low VWF and low factor VIII (8) so a product that contains both VWF and factor VIII (8) is used.
  • Some patients have a low VWF but normal factor VIII (8) so a product that only contains VWF is used.
  • Used as a treatment for patients with Type 1 VWD when DDAVP does not work well.
  • Used as a treatment for Type 2 and Type 3 VWD.

Antifibrinolytic Drugs: Tranexamic Acid or Aminocaproic Acid are often used alongside DDAVP or factor replacement.

  • These medications don’t prevent or stop bleeding. They slow or prevent the breakdown of clots.
  • Given as an oral pill or syrup.

Birth Control Pills can increase the level of VWF and factor VIII (8)

  • Used to reduce heavy menstrual bleeding.

What’s New for Von Willebrand Disease?

In January 2021, new guidelines were released about the diagnosis and treatment of VWD. 

Read more about the guidelines from the National Hemophilia Foundation.