Von Willebrand disease (VWD) is an
inherited bleeding disorder caused by a defect or deficiency of a
blood-clotting protein known as the von Willebrand factor. The von Willebrand
factor is essential for the early stages of clotting and acts like glue to help the platelets stick
together and form a blood clot. The
von Willebrand factor (VWF) is one of a dozen proteins in the body that
contribute to blood coagulation (clot formation).
We treat our patients who have von Willibrand disease at the Boston
Hemophilia Center, a federally-funded 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 von Willebrand disease.
Hemophilia Center treatment team helps children and adults with bleeding disorders to manage their illness as independently as possible and therefore lead more normal, healthy
lives. Our patients receive the benefit of access to cutting edge treatments pioneered by our researchers.
There are three types of von Willebrand disease – Type 1, 2, and 3. Type 1 is the most common.
Von Willebrand disease symptoms
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 symptoms. Others can experience symptoms such as:
It is important to understand
that some symptoms of VWD may resemble those of other more common medical
problems. Because some of these symptoms can also point to other conditions,
it’s important to have your child evaluated by a qualified medical professional
for an accurate diagnosis and prompt treatment.
Von Willebrand disease causes
VWD is an inherited disorder. The gene responsible for
the low levels of von Willebrand factor is inherited, or passed down, from a
parent. A parent with the gene for VWD has a 50 percent chance of passing the
gene on to his or her children.
If your child is suspected of having VWD, he or she will be referred to a hematologist, a doctor with
specialized training in treating blood disorders. Blood tests will be completed to measure the levels and function of VWF in the blood, including:
Other 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.
The approach to treatment
depends on the VWD type and severity and the individual’s bleeding symptoms.
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:
Your child is
an individual, and your von Willebrand disease treatment team will take many
factors into account to ensure the best treatment and follow-up plan for your
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 hemophilia and other bleeding disorders.
Participation in a clinical trial is
always completely 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
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 patients
with bleeding disorders.