Frequently Asked Questions

  • QUESTIONS ABOUT BLEEDING AND CLOTTING
  • QUESTIONS ABOUT VWD
  • QUESTIONS ABOUT TREATMENT

QUESTIONS ABOUT BLEEDING & CLOTTING

What is blood clotting?
Blood clotting is the process that occurs when a blood vessel has been damaged and the body forms a plug, or blood clot, made up of platelets, fibrin, and clotting factors to repair the injured blood vessel. This process is also known as hemostasis.1,2
What is a bleeding disorder?
A bleeding disorder is a disease in which the body can’t form blood clots properly or quickly enough in order to stop bleeding. von Willebrand disease (VWD) is an inherited bleeding disorder.2 In most cases, a person inherits VWD from a parent; in rare cases, VWD can be acquired. 1 Learn more about von Willebrand disease.
What is menorrhagia?
Menorrhagia is bleeding during the menstrual cycle that is heavier than normal or lasts longer than normal. It is a common symptom of women with VWD.3

QUESTIONS ABOUT VWD

What is von Willebrand Disease?
VWD is a disease in which a protein called von Willebrand factor (VWF) in a patient’s blood is deficient or defective.3

There are three main types of VWD1:

  • Type 1 VWD is a form of VWD in which the VWF is present at lower than normal levels, affecting blood clotting. Type 1 can be mild, moderate, or severe. Stimate Nasal Spray is indicated for use in patients with mild-to-moderate Type 1 VWD.
  • Type 2 VWD is a form of VWD in which the VWF does not work properly, affecting blood clotting. There are 4 subtypes of Type 2 VWD: Type 2A, 2B, 2M, and 2N.
  • Type 3 VWD is the most severe form of VWD, in which the VWF is almost completely absent from the blood.
What are the most common symptoms of von Willebrand disease?
Symptoms include easy bruising; nosebleeds; menorrhagia (heavy or prolonged menstrual periods); heavy bleeding after a cut; and prolonged or heavy bleeding after minor surgery, such as having a tooth pulled.1 Check your symptoms here.
Is von Willebrand disease the same thing as hemophilia?
No. Both VWD and hemophilia are bleeding disorders, but VWD is caused by a deficient or defective protein in the blood called von Willebrand factor. Hemophilia refers to bleeding disorders caused by low levels of a type of protein in the blood called factor VIII (hemophilia A) or factor IX (hemophilia B).1 While hemophilia may be more commonly known, VWD is actually a much more common disease. In fact, VWD is the most common inherited bleeding disorder in humans, affecting up to 1% of the general population.4
How do people get VWD?
In most cases, a person inherits VWD from a parent. In rare cases, VWD can be acquired, appearing in someone whose parents are not affected.1 You can create your own family tree using our Family Tree Builder to track the incidence of VWD in your family.
How can I find out if I have VWD?
If you have any of the symptoms of VWD (for example, easy bruising or frequent nosebleeds), you should get tested. It is best to see a hematologist—a doctor who specializes in testing for and treating bleeding disorders—to make or rule out a diagnosis of VWD.5 Learn more about getting tested for VWD.
If I have VWD, can I exercise or play sports?
Every sport or exercise program carries some risk of injury. But if you are fully prepared and know what to do in case of an injury, the benefits can outweigh the risks. Print out Activities to Enjoy and refer to this handy guide to recommended physical activities for people with VWD. (Before you begin any physical activity, always consult with a doctor or Hemophilia Treatment Center to make sure the activity is right for you.)

QUESTIONS ABOUT TREATMENT

What is a hematologist?
A hematologist is a doctor who specializes in diseases of the blood.3 If you suspect you have VWD, you should see a hematologist to get tested. Learn more about getting tested for VWD.
Should I keep track of bleeding episodes and medication use?
Yes. Recording your bleeds and medication use can help guide both you and your doctor in determining treatment. It can also help predict future bleeds. Our handy Online Bleeding Journal can help you keep track of bleeding symptoms, doctor appointments, medication, diet, and exercise. Just log onto AllAboutBleeding-US.com [links to www.allaboutbleeding-us.com] and register to start using this convenient interactive tool.
Is there a cure for VWD?
There is currently no cure for VWD, but the disease can be controlled with treatment. Learn more about treatment options for VWD.
What are the treatment options for VWD?
If you have been diagnosed with VWD, either the von Willebrand factor in your blood does not work properly, or there is not enough of it. To treat this condition, you need to replace the VWF that is missing or ineffective.5 Learn more about treatment options.
What is von Willebrand factor (VWF)?
VWF is the clotting protein that is deficient or missing in people with VWD. VWF is either present at lower than normal levels or does not work properly.5
What is von Willebrand factor (VWF) concentrate?
VWF concentrate is a product made from human plasma for use in the treatment of VWD; it also contains a protein called factor VIII.5
What is desmopressin?
Desmopressin acetate is a synthetic drug that is a copy of a natural hormone, vasopressin. It acts by releasing von Willebrand factor stored in the lining of the blood vessels. Two brands of desmopressin acetate are Stimate Nasal Spray, which is a high-concentration nasal spray, and DDAVP, which comes in an injectable form.6

NOTE: Stimate Nasal Spray is a high-concentration nasal spray. A low-concentration nasal spray is also available, but is used to treat diabetes insipidus and bed-wetting. The low-concentration nasal spray is NOT effective in treating VWD. People with VWD should only receive the high-concentration form of desmopressin.1 Be sure to ask for Stimate Nasal Spray by name.

What is Stimate Nasal Spray?
Stimate is a brand of high-concentration desmopressin acetate (intranasal) distributed by CSL Behring. Stimate Nasal Spray is approved for treatment of mild-to-moderate type 1 VWD. Learn more about Stimate Nasal Spray.
How does Stimate Nasal Spray work?
Stimate Nasal Spray works by triggering the body to release stored von Willebrand factor (VWF) from the cells lining the blood vessels into the bloodstream so it can help form the clots needed to stop bleeding.6 Learn more about what to expect with Stimate Nasal Spray.
What evidence is there that Stimate Nasal Spray works?
Clinical studies show that desmopressin, the active component of Stimate Nasal Spray, is an effective treatment for people with Type 1 von Willebrand disease (VWD).1
What possible side effects should I know about?
While most side effects associated with doses of Stimate Nasal Spray were mild or moderate in intensity, there is some important information you need to be aware of.1

Fluid Retention/Hyponatremia — While using this treatment, your body will hold excess fluid. In some cases, too much fluid could “dilute” the salts in your body. This could cause your body’s salt level to become very low, a condition called hyponatremia. Very rarely, cases of hyponatremia have been reported from worldwide experience in patients treated with Stimate Nasal Spray. Children and the elderly are most at risk for this side effect.

Other Possible Side Effects include facial flushing, nasal congestion, runny nose, nosebleed, sore throat, cough, and upper respiratory infections.

What is Humate-P?
Humate-P (Antihemophilic Factor/von Willebrand Factor Complex [Human]) is a product made from human plasma, used in the treatment of VWD. Humate-P contains the von Willebrand factor along with another protein called factor VIII. It is also called VWF concentrate or replacement therapy.1 Learn more about Humate-P.

Please see important safety information for Humate-P below.

How does Humate-P work?
Humate-P is a factor replacement therapy that works by replacing the VWF that is missing or ineffective in your plasma. Humate-P can be used in all types of VWD for the treatment of bleeding episodes and the prevention of bleeding during and after all surgical procedures.7-11 Learn more about Humate-P.

Please see important safety information for Humate-P below.

How effective is Humate-P?
Humate-P is a clinically proven treatment that is the most prescribed factor replacement therapy of its kind with the longest history of success.7

Please see important safety information for Humate-P below.

What are the possible side effects that I should be aware of when being treated with Humate-P?
In patients receiving Humate-P in clinical studies for treatment of VWD, the most commonly reported side effects 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 and injection-site bleeding, and epistaxis (nosebleed).

Please see important safety information for Humate-P below.

How often should I use Stimate Nasal Spray?
The convenient nasal spray pump provides an efficient, reliable way to administer your medication. If Stimate Nasal Spray is used before surgery, your hematologist will determine how far in advance of the procedure it should be administered. Your doctor can determine the need for repeat use of Stimate Nasal Spray by monitoring your condition and through laboratory tests. Use Stimate Nasal Spray exactly as prescribed. Do not use it more frequently without your doctor's approval. If bleeding is not controlled after using the spray, contact your doctor. Please see full prescribing information or go to Treatment With Stimate Nasal Spray for more information.
Can children use Stimate Nasal Spray?
Yes. Stimate Nasal Spray has been proven safe and effective in children from 11 months of age to 12 years of age. Use in infants and children will require careful fluid intake restriction. Excessive water intake can cause hyponatremia (an abnormally low level of sodium in the blood) and water intoxication. Please see full prescribing information for more information.
My doctor said I have severe Type 1 VWD. Can I use Stimate Nasal Spray?
Stimate Nasal Spray is approved for use in patients with mild-to-moderate Type 1 VWD but not for use in patients with severe Type 1 VWD1. Talk with your doctor about Humate-P, which might be a treatment option. Humate-P is a replacement therapy that can be used in all types of VWD for the treatment of bleeding episodes and the prevention of bleeding during and after all surgical procedures. If you feel that Stimate Nasal Spray or other treatments do not adequately control your bleeding; if you have Types 2 or 3 VWD; or if you have severe Type 1 VWD, Humate-P might be the right option for you.7-11 Learn more about Humate-P.
What is a bleeding disorder treatment center?
More commonly known as a Hemophilia Treatment Center or HTC, there are about 150 of these federally funded treatment centers.3 HTCs offer comprehensive health management and prevention services to people with bleeding disorders. Go to our HTC locator to find the center nearest you.
Do hemophilia treatment centers (HTCs) help people with VWD?
Yes. HTCs offer comprehensive health management and prevention services to people with bleeding disorders, including VWD. Go to our HTC locator to find the center nearest you.

References:

  1. National Heart, Lung, and Blood Institute. The Diagnosis, Evaluation, and Management of von Willebrand Disease. Bethesda, MD: National Institutes of Health; 2007. NIH Publication No. 08-5832.
  2. National Hemophilia Foundation. What is a bleeding disorder? http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF .aspx?menuid=26&contentid=5&rptname=bleeding. Accessed September 23, 2011.
  3. National Hemophilia Foundation. Glossary. http://www.hemophilia.org/NHFWeb/ MainPgs/MainNHF.aspx?menuid=39&contentid=28.
  4. Kleinert D, Orto C, Gioia K, Hannan M. Von Willebrand Disease: a nursing perspective. J Obstet Gynecol Neonatal Nurs. 1997;26:271-276.
  5. National Hemophilia Foundation. What is von Willebrand disease? http://www.hemophilia.org/ NHFWeb/MainPgs/ MainNHF.aspx?menuid=182&contentid=47&rptname=bleeding. Accessed September 23, 2011.
  6. Mannucci PM. How I treat patients with von Willebrand disease. Blood. 2001;97(7):1915-1919.
  7. Berntorp E, Archey W, Auerswald G, et al. A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate-P®/Humate-P®: history and clinical performance. Eur J Haematol. 2008;80(Suppl 70):3-35.
  8. Gill JC, Ewenstein BM, Thompson AR, Mueller-Velten G, Schwartz BA, for the Humate-P Study Group. Successful treatment of urgent bleeding in von Willebrand disease with factor VIII/VWF concentrate (Humate-P®): use of the ristocetin cofactor assay (VWF:RCo) to measure potency and to guide therapy. Haemophilia. 2003;9(6):688-695.
  9. 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(2):224-230.
  10. Lethagen S, Kyrle PA, Castaman G, Haertel S, Mannucci PM, for the Haemate P® Surgical Study Group. von Willebrand factor/factor VIII concentrate (Haemate P®) dosing based on pharmacokinetics: a prospective multicenter trial in elective surgery. J Thromb Haemost. 2007;5(7):1420-1430.
  11. Thompson AR, Gill JC, Ewenstein BM, Mueller-Velten G, Schwartz BA; Humate-P Study Group. Successful treatment for patients with von Willebrand disease undergoing urgent surgery using factor VIII/VWF concentrate (Humate-P®). Haemophilia. 2004;10(1):42-51.
  12. Centers for Disease Control and Prevention. HTC facilities directory. https://www2a.cdc.gov/ncbddd/htcweb/dir_report/Dir_view.asp?para0=REGION&para1=0&para2=FACILITY&para3=&inactive_flag=. Accessed October 10, 2011.

Important Safety Information for 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 if you have a known hypersensitivity to desmopressin or any of the components of Stimate. Stimate should not be used by 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. See full prescribing information for a complete list of adverse reactions.

Please see full prescribing information.

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.


Important Safety Information for Humate-P

Antihemophilic Factor/von Willebrand Factor Complex (Human), Humate-P® is approved to treat and prevent bleeding in adult patients with hemophilia A (classical hemophilia). Humate-P also treats spontaneous or trauma-induced bleeding episodes in adults and children with von Willebrand disease (VWD) and prevents excessive bleeding during and after surgery in patients with mild, moderate or severe VWD. Humate-P is not known to prevent spontaneous bleeding episodes.

Do not take Humate-P if you have had extreme sensitivity or an allergic response to antihemophilic or von Willebrand factor preparations. Your doctor will monitor you for events related to abnormal blood clotting.

Humate-P is made from human blood and could contain infectious agents. The risk that these agents may transmit disease cannot be completely eliminated, but has been reduced by screening plasma donors and testing donated plasma for certain viruses, and by inactivating and/or removing viruses during manufacturing.

In studies, more than 5% of patients reported the following adverse reactions to Humate-P: allergic/anaphylactic reactions, including hives, chest tightness, rash, itching, and swelling. The most common adverse reactions after surgery were bleeding at the wound or infusion site, and nosebleeds.

Please see full prescribing information.

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.