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.
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.
Humate-P® Antihemophilic Factor/von Willebrand Factor Complex (Human)
Factor replacement therapies are medicines that replace the missing VWF in people with VWD. There are two kinds of replacement therapy: 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 effective for bleeding episodes in all types of VWD.1
Humate-P is reconstituted (mixed) and taken by injection. View a video on a simple way to mix Humate-P® using the Mix2Vial™ Needle-Free Transfer Device or view the Home Infusion Video on the 6 simple steps to self-infuse Humate-P using the Mix2Vial™ device.
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. 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 hemophilia A with Factor VIII coagulant activity levels greater than 5%. Desmopressin acetate is not indicated for the treatment of hemophilia A with Factor VIII coagulant activity levels equal to or less than 5%, or for the treatment of hemophilia B, or in patients who have Factor VIII antibodies.
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 I) 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 reduce the potential for water intoxication or hyponatremia, especially in the young and the elderly, patients using desmopressin may be advised to reduce fluid intake, ingesting only enough to satisfy thirst. Your doctor will monitor for a 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 (sleepiness), dizziness, chest pain, palpitations and tachycardia (rapid heartbeat), and severe allergic reactions, including anaphylaxis.
Please see full prescribing information for Stimate.
Important Safety Information About Humate-P
Humate-P is indicated for treatment and prevention of bleeding in adult patients with hemophilia A (classical hemophilia). Humate-P is also indicated in adult and pediatric patients with von Willebrand disease (VWD) for (1) treatment of spontaneous and trauma-induced bleeding episodes, and (2) prevention of excessive bleeding during and after surgery. This applies to patients with severe VWD, and patients with mild and moderate VWD for whom use of desmopressin is known or suspected to be inadequate. Humate-P is not indicated for the prophylaxis of spontaneous bleeding episodes.
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 (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, and epistaxis (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.
Reference. 1. Lillicrap D, Poon M-C, Walker I, Xie F, Schwartz BA, and members of the Association of Hemophilia Clinic Directors of Canada. Efficacy and safety of the factor VIII/von Willebrand factor concentrate, Haemate-P/Humate-P: ristocetin cofactor unit dosing in patients with von Willebrand disease. Thromb Haemost. 2002;87:224-230.
Last Updated:
5/19/2010 12:48 PM