What is vonWillebrands Disease?
VonWillebrands disease, or vWD, is
an inherited disorder that impacts the blood's ability to clot properly.
As a group, bleeding disorders (including hemophilia) are rare. VonWillebrands disease is the most common inherited bleeding disorder,
affecting as much as 1% of the population or more. Unlike hemophilia,
which is much more common in boys, vWD affects boys and girls equally.
The child of a man or a woman with vWD has a 50% chance of getting
the gene. In types 1 and 2, a child inherits the gene for the disease
from one parent. In type 3, the child usually inherits the gene from
both parents. In those cases, the child will likely have severe symptoms,
even if the parents don't have any symptoms at all.
Normally, bleeding occurs when a blood
vessel is cut or torn. Platelets (small cells that float in the blood)
plug the hole in the blood vessel to stop the flow of blood. With
the help of calcium, vitamin K, and a protein called fibrinogen, the
platelets create a mesh to hold the plug in place and close the wound.
As this mesh dries, it hardens and forms a scab to protect the wound
as it heals. A substance in the blood known as von Willebrand factor
helps platelets stick to damaged blood vessels. Another function of
von Willebrand factor is to carry the important clotting protein,
called factor VIII, in the blood. People with von Willebrand disease
have a problem with one or both of these blood components.
Types of vonWillebrands Disease
Type 1
- Most common and the mildest form of vWD
- The level of von Willebrand factor in the blood is reduced
- The condition can be made worse by taking aspirin and other nonsteroidal
anti-inflammatory drugs
- Generally spontaneous bleeding does not occur
- Can have significant bleeding with trauma, surgery, or when a tooth
pulled
Type 2
- The von Willebrand factor has an abnormality
- There are four kinds of Type 2 vWD:
- Type 2A, in which the building blocks that make up the factor (called
multimers) are smaller than usual or break down too easily.
- Type 2B, in which the factor sticks to the platelets too well. This
leads to clumping of the platelets, which can cause a low platelet
number.
- Type 2M, in which the von Willebrand Factor''s ability to bind to
the platelets is impaired.
- Type 2N (Normandy) in which the von Willebrand factor has trouble
binding to Factor VIII thus leading to very similar results to a person
with Hemophilia A.
Type 3
- Type 3 patients have severe bleeding problems and have very low
von Willebrand factor and factor VIII (the protein that helps with
blood clotting).
Pseudo (platelet-type)
- Similar to Type 2B, but the defect is in the platelets instead of
in the factor.
Signs and Symptoms of von Willebrand
Disease
The symptoms of von Willebrand disease
may include:
- Bruising that's unusual in location or frequency
- Abnormal menstrual bleeding
- Bleeding in the mucous membranes, such as the gums, nose, and lining
of the gastrointestinal system
- Excessive or prolonged bleeding after a tooth is pulled or tonsils
are removed or prolonged oozing from cuts
Diagnosis
Because symptoms can be mild, vWD can be difficult
to diagnose and often goes undetected. The tests to diagnose vWD may
include:
- Bleeding time
- Factor VIII level test (also called factor VIII coagulant) - which
measures the level of factor VIII and its ability to function
- von Willebrand factor antigen test (also called factor VIII antigen)
- which measures the amount of von Willebrand factor. The disorder
is considered mild if levels are 20% to 40% of the normal amount.
It is severe if the amount is less than 10% of normal.
- Ristocetin cofactor activity test (also called factor VIII ristocetin
cofactor) - which measures how well the von Willebrand factor is working
- von Willebrand factor multimers test - which aids in classifying
the type of vWD
- Platelet function tests - which determine how well the platelets
work and help identify the type of vWD or the presence of another
disorder
These tests may need to be repeated more than
once because these levels may rise and fall over time in an individual.
Treatment
The most common treatment for von Willebrand
disease is desmopressin. This medication causes a temporary increase
in the von Willebrand levels. It can be given intravenously or intranasally
(into the nose), but may be ineffective in Type 2A or Type 2B.
Patients with Type 3 (and some with Type 2A
and 2B) will need treatment with a product such as Humate P, a medication
derived from human plasma that contains both factor VIII and von
Willebrand factor. This medication is given intravenously (into
the vein). Patients with Type 1 may also require the use of this
medication in certain situations, such as major trauma or major
surgery, although they can generally be treated with desmopressin.
For additional information:
Blood
Sisterhood
Project
Red Flag
The
National Hemophilia Foundation
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