Treatment Options for VWD
Licenced Clotting Factor Concentrates
- Human plasma derived dual factor concentrate
- Human plasma derived single factor concentrate
- Recombinant single factor concentrate
By-Passing Agents (For Inhibitors Only)
- Human plasma Activated Prothrombin Complex (aPCC)
- Recombinant rFVIIa
Adjunct Therapies
- DDAVP/Desmospressin (Type I/II, VWD)
- Fibrin Glue
- Tranexamic Acid
Adjunct Therapies
- Hormone treatment (women/girls)
- Intrauterine devices
Clinical trials non-factor replacements (prophylaxis only)
- Bi-specific antibodies
- Rebalancing therapies
- Aptamers
Desmopressin (DDAVP)
Desmospressin (DDAVP) is a synthetic drug, identical to a hormone normally found in the body. It is not used in children under the age of 2 years of age or in adults over the age of 55 years of age.
How Does it Work?
DDAVP stimulates your body to release your own stores of clotting factors Factor VIII and VWF into the blood stream. This helps to boost vWF levels for up to 12-24 hours. If necessary, the dose of DDAVP can be repeated after 12 hours. As DDAVP is asking the body to release stored FVIII and vWF, if you have used it within the last 12 hours, the body doesn’t have the chance to rebuild its stores, so repeated doses may not be as effective.
DDAVP Trial
Some people do not respond to DDAVP. Treatment centres may suggest doing a DDAVP trial to find out if you are a “responder”, a “partial responder” or a “non- responder”.
The trial generally takes 3 hours for children and 6 hours for adults and some children. Blood samples are taken before infusion and are taken again hourly after DDAVP infusion for up to four hours. Occasionally, a blood test will be needed the following day.
The response to DDAVP will be reported as one of the following three categories:
Responder: Your body responds well to DDAVP, resulting in a boost in VWF levels. DDAVP will be the treatment option for procedures and some bleeding episodes.
Partial responder: Your body responds only partially to DDAVP, resulting in a minor boost in VWF levels. This may be enough to cover minor procedures and minor bleeding episodes. An alternative treatment will be necessary for major procedures and major bleeding episodes.
Non-responder: Your body doesn’t respond to DDAVP. An alternative treatment will be necessary for all procedures and all bleeding episodes.
How is DDAVP given?
DDAVP is available as an intravenous injection which is often used in treatment centres and if needed can be given subcutaneously at home. The intravenous injection is slowly injected over thirty minutes to one hour.
Before DDAVP Administration
Please inform your nurse / doctor of the following:
- Any known reaction to this or any other medication
- If you take diuretics, medication for high blood pressure or any other medications, including over the counter medications and herbal remedies
- Pregnancy or breastfeeding
- Any significant medical problems including: heart disorder, kidney disorder, cystic fibrosis
Side Effects of DDAVP
Some people can experience flushing (reddening of the skin or face) or increased heart rate during the infusion. If this occurs, please tell your team; the DDAVP may need to be administered slower.
Other occasional side effects include:
- Headache
- Stomach pain and nausea
- Allergic reactions
- Decrease in blood pressure
In the elderly and in people with cardiovascular disease, DDAVP can cause more serious side effects and may not be recommended.
Fluid Intake
Adults should not drink more than 1 to 1.5 litres (approximately 8 to 10 cups) in the twenty-four hours following DDAVP. A child’s fluid intake is restricted to 75% of normal daily fluid requirement in the 24 hours following DDAVP. DDAVP can cause fluid to be retained by the body and so it is important to restrict fluid intake after the infusion of DDAVP. You will pass less urine in the 24 hours after the infusion. If you find you are passing little urine in 24 hours following the DDAVP treatment you should contact your Haemophilia centre. Treatment with DDAVP without reducing fluid intake may lead to fluid retention, dilution of salt in the blood, and in more severe cases, epileptic seizures. If these side effects occur, your doctor may advise an alternative treatment to DDAVP.
Tranexamic Acid (Cyklokapron)
Tranexamic acid (also known as Cyklokapron) is an anti-fibrinolytic agent. This means that it slows the breakdown of blood clots. It is used to prevent or treat bleeding from mucous membranes such as the inside of the mouth, nose, gut or womb. It may be given before dental work and for nosebleeds. It is often used in treating prolonged or heavy menstrual bleeding. It may be used alone or in combination with DDAVP and von Willebrand factor.
Factor Replacement Therapy
Factor concentrate is given into a vein to replace the missing vWF, to allow clotting to take place. There are two types of factor replacement available for vWD. The first is made from pooled human plasma (which is screened for blood-borne viruses such as HIV and hepatitis B and also treated to destroy viruses). This also contains FVIII. The newest option is recombinant von Willebrand factor (rvWF) which is engineered in a laboratory. The type of product used can be dependent on the type of bleeding that you are being treated for, as well as the type of vWD you have.
Some products also contain varying amounts of FVIII. This can be both helpful or unhelpful depending on the type of bleeding and when used for prophylaxis. This makes the choice of product a bit more complicated than for other bleeding disorders.
Factor replacement concentrates are infused (injected) into a vein at home by parents trained to treat their child, or by people themselves when they are confident enough to do it. It can also be done at the Haemophilia Treatment Centre if necessary.
Hormonal Contraceptive Therapy
Hormonal contraceptives reduce menstrual bleeding by thinning the endometrium and possibly increasing factor VIII and von Willebrand factor levels. They have an added advantage of controlling bleeding at ovulation and midcycle pain. Hormonal contraceptives currently available include the combined oral contraceptive pill (COC), transdermal contraceptive patches, oral progestogens, Progestin-only pills or implantable and Gonadotropin-releasing hormone (GnRH) analogues.
Women sometimes have side effects from using hormonal contraceptives such as nausea, headaches, dizziness, breast tenderness and mood changes. Serious side effects include high blood pressure, liver abnormalities and clots. Women with inherited bleeding disorders, however, may have a lower risk of clotting. Some of these side effects improve over the first 3 months. If the side effects continue the doctor may prescribe a different brand of hormonal contraceptive.
Intrauterine Device (IUD, e.g. Mirena®)
An IUD has been shown to be useful for reducing menstrual blood loss in women with bleeding disorders. The licensed duration of use in Ireland is five years i.e. once inserted it may stay in place for 5 years. Side effects may be irregular bleeding or spotting, especially within the first 6 months. There is a potential risk of bleeding at the time of insertion and preventative treatment with a haemostatic agent may be required.
To view the most recent treatment guidelines set out by the National Haemophilia Council (NHC) click here.
See Professor James O’Donnell’s presentation at our 2023 von Willebrand Disorder Information Day.