Treatment for von Willebrand disease (VWD) is based on the type of VWD and the severity of symptoms. The aim of therapy is to correct the clotting problem. This is usually accomplished by raising the levels of von Willebrand factor (VWF) and another protein called factor VIII in the bloodstream. Most cases of VWD are mild, so you may need treatment only if you have surgery, tooth extraction or an accident.
If you do require treatment for VWD, there are several options available.
DDAVP® (desmopressin acetate) Injection
This medicine is a synthetic copy of a natural hormone called vasopressin (vay-so-press-on), which works by helping the body release stored VWF into the bloodstream and increasing levels of factor VIII. Side effects include local injection-site reactions, facial flushing, headache and changes in blood pressure. It is mostly given to patients as an infusion (injection into a vein), but it can also be injected into the skin (subcutaneously).
Stimate® (desmopressin acetate) Nasal Spray 1.5 mg/ml
Stimate® (desmopressin acetate) Nasal Spray 1.5 mg/ml is another type of desmopressin, made as a high concentrate nasal spray.* Because it is sprayed into the nose (not injected), it can be easy to use. Stimate® is recommended for people with mild or moderate Type 1 VWD. The most common side effects are facial flushing and headache. Both Types of desmopressin acetate can also cause a build-up of extra water in the body and a shortage of sodium, an important mineral. And, if used too often over a short period of time, desmopressin acetate may lose its effect.
Learn more about Stimate®.
Please see prescribing information.
*There are two kinds of desmopressin nasal spray, low concentrate and high concentrate. The low concentrate nasal spray is used to treat diabetes insipidus and bed wetting and is not indicated for VWD. People with VWD should only receive the high concentrate nasal spray.
Replacement therapy
Medicines that fall into the category of replacement therapy contain actual VWF proteins to replace the ones that are missing. Replacement therapy is useful for major bleeding episodes in people who do not respond to desmopressin and for patients with type 2B and type 3 VWD.
There are two kinds of replacement product — factor concentrates containing VWF and cryoprecipitate. They are both derived from plasma and given by infusion. VWF concentrates are strongly preferred for safety reasons because they are treated to inactivate certain viruses, while cryoprecipitate is not.
Currently, the only VWF concentrate approved in the United States for all types of VWD and all medical procedures is Humate-P® Antihemophilic Factor/von Willebrand Factor Complex (Human). Humate-P® is heat-treated to inactivate or remove certain viruses. However, as with all plasma products, the risk of viral transmission of infectious agents such as viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) cannot be completely eliminated. Possible adverse events with Humate-P® include allergic reaction, urticaria, chest tightness, rash, pruritus and edema. Anaphylactic reactions can occur in rare instances. People with VWD should not receive high purity, plasma-derived factor VIII and recombinant factor VIII products. They do not contain vWF and are not effective in VWD.
Learn more about Humate-P®
Please see prescribing information.
Local clotting agents (antifibrinolytics)
Local clotting agents, or antifibrinolytics, do not help the body form blood clots. Instead, they help protect clots and hold them in place. They are often used in combination with other medicines. Clotting agents can be used to stop bleeding in the mouth, nose, or gastrointestinal tract. They can also be used to help control heavy bleeding during the menstrual period. Clotting agents can be taken as an injection or by mouth (via tablet, capsule, elixir or mouthwash).
Birth control pills
Birth control pills, or oral contraceptives, can increase VWF levels, though how they do this is not well-understood how they. Oral contraceptives can be useful for women with heavy menstrual bleeding.
As with any medical treatment, please discuss your VWD treatment questions with a doctor, nurse, or pharmacist who is a bleeding disorder specialist.
Important Safety Information About Stimate®
Stimate® Nasal Spray is indicated for patients with mild to moderate classic type 1 von Willebrand disease (VWD) with Factor VIII levels greater than 5%. Stimate® Nasal Spray is not indicated for the treatment of severe classic VWD (type 1) and when there is evidence of an abnormal molecular form of Factor VIII antigen.
Stimate® Nasal Spray is contraindicated in individuals with a known hypersensitivity to desmopressin or any of the components of Stimate®. Stimate® should not be used in patients with type 2B VWD, since platelet aggregation may be induced. Stimate® Nasal Spray is for intranasal use only.
To decrease the potential for hyponatremia or water intoxication, especially in the young and the elderly, patients using desmopressin who are not in need of antidiuretic hormone for its antidiuretic effect should be cautioned to reduce fluid intake, ingesting only enough to satisfy thirst. Such patients should be monitored for the rare occurrence of an extreme decrease in plasma osmolality that could result in seizures, possibly leading to coma.
Other adverse reactions reported with use of injectable and/or intranasal desmopressin acetate include headache, nausea, somnolence, dizziness, chest pain, palpitations and tachycardia, and severe allergic reactions, including anaphylaxis. See full prescribing information for a complete list of adverse reactions.
Please see full prescribing information for Stimate®.
Important Safety Information About Humate-P®
Humate-P® is contraindicated in individuals with a history of anaphylactic or severe systemic response to antihemophilic factor or von Willebrand factor preparations.
Monitor for intravascular hemolysis and decreasing hematocrit values in patients with A, B, and AB blood groups who are receiving large or frequent doses. Also monitor VWF:RCo and FVIII levels in VWD patients, especially those undergoing surgery.
Thromboembolic events have been reported in VWD patients receiving coagulation factor replacement. Caution should be exercised and antithrombotic measures considered, particularly in patients with known risk factors for thrombosis.
Humate-P® is derived from human plasma. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.
In patients receiving Humate-P® in clinical studies for treatment of VWD, the most commonly reported adverse reactions observed by >5% of subjects are allergic-anaphylactic reactions, including urticaria, chest tightness, rash, pruritus, and edema. For patients undergoing surgery, the most common adverse reactions are postoperative wound and injection-site bleeding, and epistaxis.
The most commonly reported adverse reactions in patients receiving Humate-P® are allergic-anaphylactic reactions, including urticaria (hives), chest tightness, rash, pruritus (itching), and edema (swelling). For patients undergoing surgery, the most common adverse reactions are postoperative wound or injection-site bleeding, or nosebleed.
Please see full prescribing information for Humate-P®.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.
Last Updated:
3/18/2010 1:43 PM