Materiały źródłowe

• #1 Von Willebrand disease – Symptoms & causes – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/von-willebrand-disease/symptoms-causes/syc-20354978

  Von Willebrand disease can’t be cured. But with treatment and self-care, most people with this disease can lead active lives. […] Rick ME. Von Willebrand disease: Treatment of minor bleeding and routine care. […] Rick ME. Von Willebrand disease: Treatment of major bleeding and major surgery. […] Connell NT, et al. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease.

• #2 Treatment of patients with von Willebrand disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3262353/

  Von Willebrand disease (vWD) is the most common hereditary bleeding disorder. The aim of therapy is to correct the dual hemostatic defect, due to defective platelet adhesion-aggregation and abnormal coagulation due to Factor VIII (FVIII) deficiency. The choice of treatment depends on a number of factors, including the severity of the bleed, the procedure planned, the subtype and severity of the disease and the age and morbidity of the patient. Desmopressin (DDAVP) is the treatment of choice for type 1 vWD as it increases endogenous release of FVIII and von Willebrand factor (vWF) and is also used in some subtypes of type 2 vWD. In those patients in whom DDAVP is ineffective or contraindicated, levels can be restored by infusing vWF:FVIII concentrates. The role of antifibrinolytic treatment is an important adjunct to replacement therapy during minor or major surgery involving mucosal surfaces. The dosing and timing of vWF:FVIII concentrates is important depending on the nature of the surgical procedure. The role of secondary prophylaxis needs to be further defined.

• #3 Principles of care for the diagnosis and treatment of von Willebrand disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3640108/

  Von Willebrand disease is a common autosomal inherited bleeding disorder caused by quantitative or qualitative defects of von Willebrand factor, a multi-adhesive protein that binds platelets to exposed subendothelium and carries factor VIII in circulation. […] The aim of treatment is to correct the dual defect of hemostasis caused by the abnormal/reduced von Willebrand factor and the concomitant deficiency of factor VIII. Desmopressin is the treatment of choice for type 1 von Willebrand disease patients with factor VIII and von Willebrand factor levels of 10 U/dL or over who have proved responsive to a test-infusion with the compound. […] Von Willebrand factor/factor VIII concentrates are needed when desmopressin is ineffective (mainly type 2 and 3 von Willebrand disease). […] Desmopressin (DDAVP) and replacement therapy with VWF/FVIII concentrate or VWF concentrates devoid of FVIII are the mainstay of treatment.

• #4

  https://haematologica.org/article/view/6652

  Von Willebrand disease is a common autosomal inherited bleeding disorder caused by quantitative or qualitative defects of von Willebrand factor, a multi-adhesive protein that binds platelets to exposed subendothelium and carries factor VIII in circulation. […] The aim of treatment is to correct the dual defect of hemostasis caused by the abnormal/reduced von Willebrand factor and the concomitant deficiency of factor VIII. Desmopressin is the treatment of choice for type 1 von Willebrand disease patients with factor VIII and von Willebrand factor levels of 10 U/dL or over who have proved responsive to a test-infusion with the compound. Von Willebrand factor/factor VIII concentrates are needed when desmopressin is ineffective (mainly type 2 and 3 von Willebrand disease). […] Desmopressin (DDAVP) and replacement therapy with VWF/FVIII concentrate or VWF concentrates devoid of FVIII are the mainstay of treatment.

• #5 Treatment of patients with von Willebrand disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3262353/

  Von Willebrand disease (vWD) is the most common hereditary bleeding disorder. The aim of therapy is to correct the dual hemostatic defect, due to defective platelet adhesion-aggregation and abnormal coagulation due to Factor VIII (FVIII) deficiency. The choice of treatment depends on a number of factors, including the severity of the bleed, the procedure planned, the subtype and severity of the disease and the age and morbidity of the patient. Desmopressin (DDAVP) is the treatment of choice for type 1 vWD as it increases endogenous release of FVIII and von Willebrand factor (vWF) and is also used in some subtypes of type 2 vWD. In those patients in whom DDAVP is ineffective or contraindicated, levels can be restored by infusing vWF:FVIII concentrates. The role of antifibrinolytic treatment is an important adjunct to replacement therapy during minor or major surgery involving mucosal surfaces. The dosing and timing of vWF:FVIII concentrates is important depending on the nature of the surgical procedure. The role of secondary prophylaxis needs to be further defined.

• #6

  https://childrenswi.org/publications/teaching-sheet/hematology/1466-von-willebrand-disease

  Treatment for VWD is based on which type your child has. Most cases of VWD are mild. In that case, your child may only need treatment after surgery, dental work, or trauma. People with type 3 VWD may need emergency treatment to stop bleeding. […] Treatments may include: A man-made hormone called desmopressin. This hormone is taken by injection or as a nasal spray. It makes the body release more Von Willebrand factor and factor VIII into the blood. Desmopressin works for most people who have type 1 VWD and for some who have type 2. Therapy to replace Von Willebrand factor. Factor VIII and Von Willebrand factor are injected into a vein in the arm. This treatment may be used if your child: Can’t take desmopressin or needs treatment for a long time. Has type 1 VWD that does not respond to desmopressin. Has type 2 or type 3 VWD. Antifibrinolytic medicines. These medicines help keep blood clots from breaking down. They are mostly used to stop bleeding after minor surgery, having a tooth pulled, or an injury. They may be used alone or with desmopressin and replacement therapy. Fibrin glue. This is put right on a wound to stop bleeding.

• #7 Diagnosis and Management of Von Willebrand Disease: Guidelines for Primary Care | AAFP

  https://www.aafp.org/pubs/afp/issues/2009/1201/p1261.html

  Von Willebrand disease is an inherited condition characterized by deficiency of von Willebrand factor, which is essential in hemostasis. […] Treatment is tailored to the subtype of the disease: increasing plasma concentration of von Willebrand factor by releasing endogenous stores with desmopressin or replacing nonexistent or ineffective von Willebrand factor by using human plasmaderived, viral-inactivated concentrates; treatment is often combined with hemostatic agents that have mechanisms other than increasing von Willebrand factor. […] There are three main approaches to the treatment of VWD, used individually or in combination. These approaches include increasing plasma concentration of VWF by releasing endogenous VWF stores through stimulation of endothelial cells with desmopressin (DDAVP); replacing VWF by using human plasmaderived, viral-inactivated concentrates; and promoting hemostasis using hemostatic agents with mechanisms other than increasing VWF. Regular prophylaxis is seldom required, and treatment is initiated before planned invasive procedures or in response to bleeding.

• #8 Von Willebrand disease – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/von-willebrand-disease/diagnosis-treatment/drc-20354984

  Even though von Willebrand disease has no cure, treatment can help prevent or stop bleeding episodes. Your treatment depends on: […] Your doctor might suggest one or more of the following treatments to increase your von Willebrand factor, strengthen blood clots or control heavy menstrual bleeding: […] Desmopressin. This medication is available as an injection (DDAVP). It’s a synthetic hormone that controls bleeding by stimulating your body to release more of the von Willebrand factor stored in the lining of your blood vessels. […] Many doctors consider desmopressin (DDAVP) the first treatment for managing von Willebrand disease. It can be used before minor surgical procedures to help control bleeding. You might be given a trial of desmopressin to make sure it’s effective for you. […] Replacement therapies. These include infusions of concentrated blood-clotting factors containing von Willebrand factor and factor VIII. Your doctor might recommend them if DDAVP isn’t an option for you or has been ineffective.

• #9 Medications for von Willebrand Disease | NYU Langone Health

  https://nyulangone.org/conditions/von-willebrand-disease-in-children/treatments/medications-for-von-willebrand-disease

  Desmopressin, also known as DDAVP, is a synthetic hormone that stimulates the release of von Willebrand factor from the cells that line blood vessels. This medication, available as a nasal spray or injection, is often recommended for children or adolescents with type 1 or certain forms of type 2 von Willebrand disease to control bleeding before surgery, during menstrual periods, or during a prolonged nosebleed. […] Your childs doctor may recommend replacing von Willebrand factor through a vein as an intravenous (IV) treatment. This therapy may be used regularly to prevent or control bleeding in children who do not respond to DDAVP or have more severe bleeding episodes, which can occur in those with type 3 von Willebrand disease. It may also be given before surgical procedures. […] Clot stabilizers, also known as antifibrinolytics, slow down the formation of fibrin, a substance that breaks down blood clots. Antifibrinolytic medications, such as aminocaproic acid, may be given to your child by mouth or through an IV infusion to stop bleeding from a surgical procedure or injury. Your child also may receive an oral solution for rinsing the mouth before or after a dental procedure.

• #10 Principles of care for the diagnosis and treatment of von Willebrand disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3640108/

  Von Willebrand disease is a common autosomal inherited bleeding disorder caused by quantitative or qualitative defects of von Willebrand factor, a multi-adhesive protein that binds platelets to exposed subendothelium and carries factor VIII in circulation. […] The aim of treatment is to correct the dual defect of hemostasis caused by the abnormal/reduced von Willebrand factor and the concomitant deficiency of factor VIII. Desmopressin is the treatment of choice for type 1 von Willebrand disease patients with factor VIII and von Willebrand factor levels of 10 U/dL or over who have proved responsive to a test-infusion with the compound. […] Von Willebrand factor/factor VIII concentrates are needed when desmopressin is ineffective (mainly type 2 and 3 von Willebrand disease). […] Desmopressin (DDAVP) and replacement therapy with VWF/FVIII concentrate or VWF concentrates devoid of FVIII are the mainstay of treatment.

• #11

  https://haematologica.org/article/view/6652

  Von Willebrand disease is a common autosomal inherited bleeding disorder caused by quantitative or qualitative defects of von Willebrand factor, a multi-adhesive protein that binds platelets to exposed subendothelium and carries factor VIII in circulation. […] The aim of treatment is to correct the dual defect of hemostasis caused by the abnormal/reduced von Willebrand factor and the concomitant deficiency of factor VIII. Desmopressin is the treatment of choice for type 1 von Willebrand disease patients with factor VIII and von Willebrand factor levels of 10 U/dL or over who have proved responsive to a test-infusion with the compound. Von Willebrand factor/factor VIII concentrates are needed when desmopressin is ineffective (mainly type 2 and 3 von Willebrand disease). […] Desmopressin (DDAVP) and replacement therapy with VWF/FVIII concentrate or VWF concentrates devoid of FVIII are the mainstay of treatment.

• #12 Patient education: von Willebrand disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/von-willebrand-disease-beyond-the-basics

  If you learn that you need a medical procedure or surgery, including dental surgery, you should discuss your VWD with everyone involved in the procedure ahead of time (hematologist, dentist, surgeon, anesthesiologist). Your type of VWD, previous history of bleeding, and options to control bleeding will be discussed. […] People with VWD will generally need closer-than-average monitoring during and after surgery. […] Some people will not need any additional treatment, depending on their type of VWD and the type of procedure. […] DDAVP – Some people can be treated with a medicine called DDAVP (also called desmopressin). This medicine helps your body release extra von Willebrand factor (VWF) into the bloodstream. However, this effect lasts only for 6 to 24 hours and may not be sufficient for some people. DDAVP can be given as a nasal spray, injected under the skin, or infused through an intravenous line. Doses are given 24 hours apart and should generally not be used for more than three days in a row.

• #13 Treatment of von Willebrand disease | Hemophilia

  https://www.hemophilia.ca/treatment-of-von-willebrand-disease/

  Treatment of von Willebrand disease […] No. Minor bleeding episodes associated with VWD often do not require medical treatment. For example: […] However, sometimes medical treatment is necessary. The type of treatment depends in part on the type of VWD a person has. […] Therefore, the best place for a person with VWD to get treatment is a bleeding disorder treatment centre. […] Once a proper diagnosis has been made, and a treatment plan has been organized, the doctors at the bleeding disorder treatment centre can work with the individual and his/her family physician to provide care. […] Desmopressin is the preferred treatment for Type 1 VWD. It can be taken in three different ways. […] Desmopressin is effective for many people with Type 1 VWD. However, different people respond to desmopressin in different ways. Therefore, a doctor needs to do tests to find out each individual’s response to the drug. Ideally, these tests are done before any urgent need for the drug, such as surgery.

• #14 Diagnosis and Treatment of von Willebrand Disease and Rare Bleeding Disorders

  https://www.mdpi.com/2077-0383/6/4/45

  Effective treatment in VWD requires the correction of the dual haemostatic defect in case of bleeding or before an intervention. Current therapeutic strategies involve either elevation of the plasma concentrations through the release of endogenous VWF from endothelial cells with desmopressin (DDAVP) or replacement therapy of VWF with human plasma-derived (pd) low-purity FVIII-VWF concentrates or a high-purity VWF product. Other treatments can be considered additional to these two modalities. […] DDAVP (1-deamino-8-d-arginin-vasopressin), a synthetic analog of the antidiuretic hormone vasopressin, V2 agonist, increases VWF and FVIII in plasma without significant side effects. Other important advantages of the drug are its safety from the risk of transmitting blood-borne viruses and its low cost. DDAVP can be administered intravenously (0.3 mcg/kg diluted in 50–100 mL saline and infused over 30 min), subcutaneously (0.3 mcg/kg), or intranasally (fixed doses of 300 mcg in adults and 150 mcg in children). This treatment increases circulating FVIII/VWF three to five times above basal levels within 30–60 min; high plasma FVIII/VWF concentrations last for 6–8 h. DDAVP dose can be repeated after 12 to 24 h, depending on the individual response, which is influenced by various factor, including genotype and phenotype.

• #15 Treatment for von Willebrand Disease > Von Willebrand Disease > Bleeding Disorders > HoG Handbook > Hemophilia of Georgia

  https://www.hog.org/handbook/article/1/7/treatment-for-von-willebrand-disease

  Von Willebrand Disease can be treated in several ways. The doctor at the HTC can check to see which treatment will work the best. Treatment for VWD One medicine that is used is DDAVP (desmopressin acetate). This drug causes von Willebrand factor that is stored in the body to be released into the bloodstream. Because it does not work for everyone, the doctor will first check the effect of DDAVP by giving the patient a dose when she or he is not bleeding. The doctor will then measure the von Willebrand factor level in the blood to see if it went up enough to stop bleeding. DDAVP can cause side effects low sodium and possibly seizures so doctors may not want to use it in elderly people or children under age five. […] Most people use a form of DDAVP that is sprayed into the nose. It is known by the brand name Stimate. From the nose, it is absorbed into the blood. DDAVP can also be injected directly into the blood through a vein or given as a shot under the skin.

• #16 von Willebrand Disease | Hemophilia Treatment Center | ECU

  https://ecuphysicians.ecu.edu/hemophilia-treatment-center/von-willebrand-disease/

  Even though von Willebrand disease is a lifelong condition with no cure, treatment can help prevent or stop bleeding episodes. Your treatment depends on the type and severity of your condition, how you’ve responded to previous therapy, and your other medications and conditions. Your doctor may suggest one or more of the following treatments to increase your von Willebrand factor, strengthen blood clots or, in women, control heavy menstrual bleeding: […] Stimate/Desmopressin This medication is available as an injection (DDAVP) or nasal spray (Stimate). It’s a synthetic hormone, similar to the natural hormone vasopressin. It controls bleeding by stimulating your body to release more von Willebrand factor already stored in the lining of your blood vessels. DDAVP is usually effective in people with type 1 and some subtypes of type 2 disease.

• #17 Von Willebrand Disease Treatment Guidelines | NBDF

  https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-266-masac-recommendations-regarding-the-treatment-of-von-willebrand-disease

  The following are current recommendations for treating bleeding in individuals with VWD. They are adapted from the ASH, ISTH, NHF, WFH 2021 VWD guidelines. (3) […] […] 1. Persons with type 1, 2A, 2M, and 2N VWD may be treated with the synthetic agent desmopressin (DDAVP Injectable or Stimate Nasal Spray for Bleeding, 1.5 mg/ml) if they have been shown by a DDAVP trial to be responsive. This is particularly important for patients with type 1 VWD and VWF =30% and types 2A, 2M, and 2N who may not have sufficient response. Response should be assessed one and four hours after DDAVP; the one-hour assessment is particularly important for patients suspected of having type 1C VWD. A desmopressin response requires an increase of at least 2 times the baseline VWF activity level and a sustained increase of both VWF and factor VIII (FVIII):C levels 0.50 IU/mL for at least 4 hours. […]

• #18 Patient education: von Willebrand disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/von-willebrand-disease-beyond-the-basics

  The only way to know if DDAVP will work for you is to do a test after your diagnosis is established and when you are not bleeding or otherwise sick. During the test, you are given a dose of DDAVP and your blood is checked before and after the dose to see if your VWF and factor VIII levels increase sufficiently, usually near or into the normal range. […] If you are helped by the DDAVP nose spray, it is important to use the form intended for VWD (there are other forms intended to treat other conditions, such as fluid disorders or bedwetting, which are not effective for treating VWD). […] It is also important to limit fluid intake for 12 to 24 hours while taking DDAVP, as DDAVP can cause water retention that can lead to serious symptoms, including seizures. Some older people or people with heart disease or a history of stroke may also need to avoid using DDAVP. DDAVP is generally avoided in patients <2 years of age.

• #19 Treatment of von Willebrand Disorder – Irish Haemophilia Society

  https://haemophilia.ie/about-bleeding-disorders/vwd-2/treatment/

  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. […] 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 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.

• #20 von Willebrand disease – Wikipedia

  https://en.wikipedia.org/wiki/Von_Willebrand_disease

  For patients with VWD type 1 and VWD type 2A, desmopressin is available as different preparations, recommended for use in cases of minor trauma, or in preparation for dental or minor surgical procedures. Desmopressin stimulates the release of VWF from the Weibel-Palade bodies of endothelial cells, thereby increasing the levels of VWF (as well as coagulant factor VIII) three- to five-fold. Desmopressin is also available as a preparation for intranasal administration (Stimate) and as a preparation for intravenous administration. Desmopressin is contraindicated in VWD type 2b because of the risk of aggravated thrombocytopenia and thrombotic complications. Desmopressin is probably not effective in VWD type 2M and is rarely effective in VWD type 2N. It is totally ineffective in VWD type 3. […] For women with heavy menstrual bleeding, estrogen-containing oral contraceptive medications are effective in reducing the frequency and duration of the menstrual periods. Estrogen and progesterone compounds available for use in the correction of menorrhagia include ethinylestradiol, levonorgestrel, drospirenone and cyproterone. Administration of ethinylestradiol diminishes the secretion of luteinizing hormone and follicle-stimulating hormone from the pituitary, leading to stabilization of the endometrial surface of the uterus.

• #21 Treatment of von Willebrand disease | Hemophilia

  https://www.hemophilia.ca/treatment-of-von-willebrand-disease/

  Desmopressin is not often recommended for Type 2 VWD. […] However, some individuals with Type 2A VWD have a good response to desmopressin. Therefore, it is recommended that, at the time of diagnosis, tests be done to measure a person’s response to desmopressin. […] Cryoprecipitate is a blood component made from plasma, containing VWF and factor VIII. It was commonly used to treat VWD in the past. However, because there is no method to kill viruses in cryoprecipitate, it is no longer recommended. […] Currently, techniques are being developed to treat cryoprecipitate for viruses. When they are proven effective, cryoprecipitate may once again be used as a treatment for VWD.

• #22 Treatment of patients with von Willebrand disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3262353/

  DDAVP does not have any therapeutic use in type 3 vWD, and these patients must be treated with a vWF-containing concentrate. […] For those patients in whom DDAVP is ineffective or contraindicated, vWF and FVIII levels can be restored by infusion of concentrates, which may contain both FVIII and vWF or very high-purity vWF with minimum or no FVIII. […] Management of mucosal bleeding, menorrhagia or prolonged oral bleeding after dental extraction involves the administration of antifibrinolytic amino acids (eg, tranexamic acid, epilson aminocapric acid). These agents inhibit the conversion of plasminogen to plasmin, inhibiting fibrinolysis and thereby helping to stabilize clots that have been formed. […] Platelet transfusion should be considered in patients with type 3 or platelet-low vWD to control bleeding that is not responsive or poorly responsive to replacement therapy with vWF concentrate.

• #23 Patient education: von Willebrand disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/von-willebrand-disease-beyond-the-basics

  The only way to know if DDAVP will work for you is to do a test after your diagnosis is established and when you are not bleeding or otherwise sick. During the test, you are given a dose of DDAVP and your blood is checked before and after the dose to see if your VWF and factor VIII levels increase sufficiently, usually near or into the normal range. […] If you are helped by the DDAVP nose spray, it is important to use the form intended for VWD (there are other forms intended to treat other conditions, such as fluid disorders or bedwetting, which are not effective for treating VWD). […] It is also important to limit fluid intake for 12 to 24 hours while taking DDAVP, as DDAVP can cause water retention that can lead to serious symptoms, including seizures. Some older people or people with heart disease or a history of stroke may also need to avoid using DDAVP. DDAVP is generally avoided in patients <2 years of age.

• #24 Treatment of von Willebrand disease | Hemophilia

  https://www.hemophilia.ca/treatment-of-von-willebrand-disease/

  In major surgery, desmopressin alone may not be enough to control bleeding. In such a case, a person should also receive a concentrate of VWF and factor VIII. […] Desmopressin can sometimes have some mild side effects. These are: facial flushing, mild headache, nausea and abdominal cramps. […] Cyklokapron (tranexamic acid) and Amicar (aminocaproic acid) are drugs that help to hold a clot in place once it has formed. […] They can be used to hold a clot in place in mucous membranes such as: the inside of the mouth, the inside of the nose, inside the intestines (the gut), inside the uterus (the womb). […] Factor VIII/von Willebrand factor concentrate (FVIII/VWF concentrate) is the preferred treatment for: Type 3 VWD, most forms of Type 2 VWD, for serious bleeding or major surgery in all types of VWD. […] FVIII/VWF concentrate is injected into a vein. It can be administered at a clinic, doctor’s office or emergency room. Many people learn to inject it themselves at home.

• #25 Treatment of von Willebrand Disorder – Irish Haemophilia Society

  https://haemophilia.ie/about-bleeding-disorders/vwd-2/treatment/

  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. […] 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 […] 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.

• #26 Von Willebrand Disease: Types, Causes, and Symptoms

  https://www.healthline.com/health/von-willebrand-disease

  If you have von Willebrand disease, your treatment plan will vary, depending on the type of condition you have. Your doctor may recommend several different treatments. […] Your doctor may prescribe desmopressin (DDAVP), which is a drug recommended for types 1 and 2A. DDAVP stimulates the release of VWF from your bodys cells. Common side effects include a headache, low blood pressure, and fast heart rate. […] Your doctor may also recommend replacement therapy, using Humate-P or Alphanate Solvent Detergent/Heat Treated (SD/HT). These are two types of biologics, or genetically engineered proteins. Theyre developed from human plasma. They can help replace the VWF thats absent or working improperly in your body. […] Common side effects of replacement therapy with Humate-P and Alphanate SD/HT include chest tightness, rash, and swelling.

• #27 Von Willebrand disease – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/von-willebrand-disease/diagnosis-treatment/drc-20354984

  Even though von Willebrand disease has no cure, treatment can help prevent or stop bleeding episodes. Your treatment depends on: […] Your doctor might suggest one or more of the following treatments to increase your von Willebrand factor, strengthen blood clots or control heavy menstrual bleeding: […] Desmopressin. This medication is available as an injection (DDAVP). It’s a synthetic hormone that controls bleeding by stimulating your body to release more of the von Willebrand factor stored in the lining of your blood vessels. […] Many doctors consider desmopressin (DDAVP) the first treatment for managing von Willebrand disease. It can be used before minor surgical procedures to help control bleeding. You might be given a trial of desmopressin to make sure it’s effective for you. […] Replacement therapies. These include infusions of concentrated blood-clotting factors containing von Willebrand factor and factor VIII. Your doctor might recommend them if DDAVP isn’t an option for you or has been ineffective.

• #28 Von Willebrand disease – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/von-willebrand-disease/diagnosis-treatment/drc-20354984

  Another replacement therapy approved by the Food and Drug Administration for treating adults 18 and older is a genetically engineered (recombinant) von Willebrand factor product. Because recombinant factor is made without plasma, it can reduce the risk of a viral infection or allergic reaction. […] Oral contraceptives. In addition to preventing pregnancy, these drugs can help control heavy bleeding during menstrual periods. The estrogen hormones in birth control pills can boost von Willebrand factor and factor VIII activity. […] Clot-stabilizing medications. These anti-fibrinolytic medications such as aminocaproic acid (Amicar) and tranexamic acid (Cyklokapron, Lysteda) can help stop bleeding by slowing the breakdown of blood clots. Doctors often prescribe these drugs before or after a surgical procedure or tooth extraction.

• #29 von Willebrand Disease Treatment & Management: Approach Considerations, Type I von Willebrand disease, Type 2 von Willebrand disease

  https://emedicine.medscape.com/article/206996-treatment

  The main treatment options for patients with von Willebrand disease (vWD) are as follows: Desmopressin (DDAVP) – DDAVP is a synthetic analogue of the antidiuretic hormone vasopressin; it has enhanced antidiuretic activity and no pressor activity related to vasopressin. […] Recombinant von Willebrand factor (rVWF) – Indicated for on-demand treatment of minor or major hemorrhage in adults with vWD. […] Von Willebrand factor/factor VIII (vWF/FVIII) concentrates. Purified plasma-derived concentrates of vWF/FVIII are used for treatment of bleeds and for surgical prophylaxis when DDAVP is ineffective or contraindicated. […] Recombinant von Willebrand factor (Vonvendi) was approved by the US Food and Drug Administration (FDA) in 2015. Approval was based on a phase 3 trial that showed that rVWF was safe and effective in treating 192 bleeds in 22 patients with vWD and stabilized endogenous FVIII:C levels.

• #30 Treatment of von Willebrand disease | Hemophilia

  https://www.hemophilia.ca/treatment-of-von-willebrand-disease/

  In major surgery, desmopressin alone may not be enough to control bleeding. In such a case, a person should also receive a concentrate of VWF and factor VIII. […] Desmopressin can sometimes have some mild side effects. These are: facial flushing, mild headache, nausea and abdominal cramps. […] Cyklokapron (tranexamic acid) and Amicar (aminocaproic acid) are drugs that help to hold a clot in place once it has formed. […] They can be used to hold a clot in place in mucous membranes such as: the inside of the mouth, the inside of the nose, inside the intestines (the gut), inside the uterus (the womb). […] Factor VIII/von Willebrand factor concentrate (FVIII/VWF concentrate) is the preferred treatment for: Type 3 VWD, most forms of Type 2 VWD, for serious bleeding or major surgery in all types of VWD. […] FVIII/VWF concentrate is injected into a vein. It can be administered at a clinic, doctor’s office or emergency room. Many people learn to inject it themselves at home.

• #31 Von Willebrand Disease Treatment Guidelines | NBDF

  https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-266-masac-recommendations-regarding-the-treatment-of-von-willebrand-disease

  3. VWF Replacement […] Persons with type 2B and type 3 VWD, and those with type 1, 2A, 2M, and 2N who have been shown to be nonresponsive to DDAVP, should be treated with a factor VIII/VWF concentrate that is known to contain the higher molecular weight multimers of von Willebrand factor and that has been virally attenuated to eliminate transmission of HIV and hepatitis A, B, and C. Human plasma-derived products Alphanate, Humate P, and Wilate have been approved by the FDA for use in VWD. A recombinant VWF concentrate, Vonvendi, has also been approved. […] […] 4. Antifibrinolytics […] Adjunctive treatments for mucous membrane bleeding include the antifibrinolytic agents aminocaproic acid and tranexamic acid. These agents can be given orally or intravenously. […] […] 6. Prophylaxis […] In patients with VWD with a history of major and frequent bleeds, the ASH ISTH NHF WFH guideline panel suggests using long-term prophylaxis with factor replacement rather than no prophylaxis.(3) Prophylaxis in VWD is defined as a period of at least 3 months of treatment of VWF concentrate at least once weekly, or for women with HMB, use of VWF concentrate at least once per menstrual cycle. […]

• #32 von Willebrand disease – eLearning Platform

  https://elearning.wfh.org/elearning-centres/von-willebrand-disease/

  There are two types of VWF-containing concentrates. The first type is plasma derived that is purified from blood. These concentrates contain VWF and varying amounts of FVIII. The other type is recombinant, a version of VWF that is made in a laboratory and is not obtained from blood donation. These concentrates are the preferred treatment for type 3 VWD, most forms of type 2 VWD, and for serious bleeding or major surgery in all types of VWD. […] Hormone treatment, such as oral contraceptives, can be an option for some women and girls with VWD. These hormonal drugs help to reduce heavy menstrual bleeding and can also prevent pregnancy. In women who are not planning to become pregnant, an intrauterine device (IUD) may be a good option to help control heavy periods as it can last up to five years. In women with heavy periods who are trying to have a baby, hormone treatments cannot be used. Antifibrinolytic agents, DDAVP, or VWF concentrates may be effective for treating heavy menstruation.

• #33 von Willebrand disease – eLearning Platform

  https://elearning.wfh.org/elearning-centres/von-willebrand-disease/

  There are two types of VWF-containing concentrates. The first type is plasma derived that is purified from blood. These concentrates contain VWF and varying amounts of FVIII. The other type is recombinant, a version of VWF that is made in a laboratory and is not obtained from blood donation. These concentrates are the preferred treatment for type 3 VWD, most forms of type 2 VWD, and for serious bleeding or major surgery in all types of VWD. […] Hormone treatment, such as oral contraceptives, can be an option for some women and girls with VWD. These hormonal drugs help to reduce heavy menstrual bleeding and can also prevent pregnancy. In women who are not planning to become pregnant, an intrauterine device (IUD) may be a good option to help control heavy periods as it can last up to five years. In women with heavy periods who are trying to have a baby, hormone treatments cannot be used. Antifibrinolytic agents, DDAVP, or VWF concentrates may be effective for treating heavy menstruation.

• #34 Diagnosis and Treatment of von Willebrand Disease and Rare Bleeding Disorders

  https://www.mdpi.com/2077-0383/6/4/45

  For those VWD patients in whom DDAVP is either ineffective or contraindicated, replacement therapy with pd-VWF/FVIII concentrates is the treatment of choice. Current effective intermediate and high-purity VWF-FVIII concentrates are several and all plasma-derived, with a variable content of VWF and FVIII, as well as a heterogeneous multimer pattern. […] Different recommendations or national guidelines for VWD treatment are available, and useful indications for replacement therapy in spontaneous bleedings, minor and major surgeries, delivery and puerperium, development of alloantibodies are summarized. […] In contrast to patients with haemophilia, regular replacement therapy to prevent bleeding is not commonly used in those with VWD, even if its benefit has been described in several case series. In patients with severe forms of VWD, when plasma FVIII level is <5 U/dL, suffering from recurrent joint bleeding or gastrointestinal bleeding, a secondary long-term prophylaxis may represent an efficacious treatment.

• #35 Treatment for von Willebrand Disease > Von Willebrand Disease > Bleeding Disorders > HoG Handbook > Hemophilia of Georgia

  https://www.hog.org/handbook/article/1/7/treatment-for-von-willebrand-disease

  Factor concentrate is the usual treatment for people with severe Type 1, Type 2A, Type 2B, and Type 3 VWD and those whose bodies dont respond to DDAVP. People with VWD must use factor concentrate that has VWF in it. This means that they cannot use the recombinant factor products used by people with Hemophilia. Those products only contain factor VIII or factor IX. They dont have any von Willebrand factor. In the United States, these are the brands of factor concentrate that contain VWF: Alphanate SD, Humate P, Koate DVI, Wilate, and Vonvendi (the first recombinant VWF approved by the FDA). In an emergency, doctors might give a person with VWD a blood product called cryoprecipitate to stop bleeding. It contains von Willebrand factor along with other factor proteins. However, since cryoprecipitate is not treated to kill viruses, it is not recommended. It is used only if other medicines are not available at all.

• #36 VONVENDI® Treatment for Adults With von Willebrand Disease (VWD)

  https://www.vonvendi.com/

  Find a treatment specifically created for von Willebrand disease (VWD) bleeding episodes. […] VONVENDI is used to treat bleeds in adults with von Willebrand disease (VWD), an inherited bleeding disorder that makes it difficult to form blood clots. […] VONVENDI is the first and only recombinant treatment for VWD, meaning it’s made without using human blood. […] VONVENDI is also the first and only recombinant treatment for VWD that’s approved for three uses including: Routine prophylaxis in appropriate adult patients with severe type 3 VWD, On-demand to manage active bleeds, Perioperative to help prevent bleeding episodes during or after surgery. […] In certain adult patients with severe Type 3 VWD, VONVENDI is approved to reduce the frequency of bleeding episodes. […] VONVENDI is used in adults (age 18 years and older) diagnosed with von Willebrand disease to: treat and control bleeding episodes, prevent excessive bleeding during and after surgery, reduce the number of bleeding episodes when used regularly (prophylaxis) in adults with severe Type 3 von Willebrand disease receiving on-demand therapy.

• #37 About von Willebrand Disease | Von Willebrand Disease (VWD) | CDC

  https://www.cdc.gov/von-willebrand/about/index.html

  The type of treatment prescribed for VWD depends on the type and severity of the disease. For minor bleeds, treatment might not be needed. […] The most commonly used types of treatment are: […] This medicine (DDAVP) is injected into a vein to treat people with milder forms of VWD (mainly type 1). It works by making the body release more VWF into the blood. It also helps increase the level of factor VIII in the blood. […] This high-strength nasal spray (Stimate) is used to treat people with milder forms of VWD (mainly type 1). It works by making the body release more VWF into the blood. […] Recombinant VWF (such as Vonvendi) and medicines rich in VWF and factor VIII (for example, Humate P, Wilate, Alphanate, or Koate DVI) are used to treat people with more severe forms of VWD or people with milder forms of VWD who do not respond well to the nasal spray. These medicines are injected into a vein in the arm to replace the missing factor in the blood.

• #38 von Willebrand Disease Treatment & Management: Approach Considerations, Type I von Willebrand disease, Type 2 von Willebrand disease

  https://emedicine.medscape.com/article/206996-treatment

  The main treatment options for patients with von Willebrand disease (vWD) are as follows: Desmopressin (DDAVP) – DDAVP is a synthetic analogue of the antidiuretic hormone vasopressin; it has enhanced antidiuretic activity and no pressor activity related to vasopressin. […] Recombinant von Willebrand factor (rVWF) – Indicated for on-demand treatment of minor or major hemorrhage in adults with vWD. […] Von Willebrand factor/factor VIII (vWF/FVIII) concentrates. Purified plasma-derived concentrates of vWF/FVIII are used for treatment of bleeds and for surgical prophylaxis when DDAVP is ineffective or contraindicated. […] Recombinant von Willebrand factor (Vonvendi) was approved by the US Food and Drug Administration (FDA) in 2015. Approval was based on a phase 3 trial that showed that rVWF was safe and effective in treating 192 bleeds in 22 patients with vWD and stabilized endogenous FVIII:C levels.

• #39 VONVENDI® Treatment for Adults With von Willebrand Disease (VWD)

  https://www.vonvendi.com/

  Find a treatment specifically created for von Willebrand disease (VWD) bleeding episodes. […] VONVENDI is used to treat bleeds in adults with von Willebrand disease (VWD), an inherited bleeding disorder that makes it difficult to form blood clots. […] VONVENDI is the first and only recombinant treatment for VWD, meaning it’s made without using human blood. […] VONVENDI is also the first and only recombinant treatment for VWD that’s approved for three uses including: Routine prophylaxis in appropriate adult patients with severe type 3 VWD, On-demand to manage active bleeds, Perioperative to help prevent bleeding episodes during or after surgery. […] In certain adult patients with severe Type 3 VWD, VONVENDI is approved to reduce the frequency of bleeding episodes. […] VONVENDI is used in adults (age 18 years and older) diagnosed with von Willebrand disease to: treat and control bleeding episodes, prevent excessive bleeding during and after surgery, reduce the number of bleeding episodes when used regularly (prophylaxis) in adults with severe Type 3 von Willebrand disease receiving on-demand therapy.

• #40 VONVENDI® Prophylaxis Treatment for Von Willebrand Disease (VWD)

  https://www.vonvendipro.com/prophylaxis

  FOR YOUR SEVERE TYPE 3 VON WILLEBRAND DISEASE (VWD) PATIENTS RECEIVING ON-DEMAND THERAPY1 […] In a clinical trial, routine prophylaxis with VONVENDI helped reduce the number of bleeds for adults with severe Type 3 VWD who were previously treated on demand (N=10).1 […] VONVENDI [von Willebrand factor (Recombinant)] is a recombinant von Willebrand factor (rVWF) indicated for use in adults (age 18 and older) diagnosed with von Willebrand disease (VWD) for on-demand treatment and control of bleeding episodes; perioperative management of bleeding; routine prophylaxis to reduce the frequency of bleeding episodes in patients with severe Type 3 von Willebrand disease receiving on-demand therapy.1 […] As the first and only approved recombinant prophylaxis treatment for VWD, VONVENDI [von Willebrand factor (Recombinant)] provides bleed protection

• #41 VONVENDI® Treatment for Adults With von Willebrand Disease (VWD)

  https://www.vonvendi.com/

  Find a treatment specifically created for von Willebrand disease (VWD) bleeding episodes. […] VONVENDI is used to treat bleeds in adults with von Willebrand disease (VWD), an inherited bleeding disorder that makes it difficult to form blood clots. […] VONVENDI is the first and only recombinant treatment for VWD, meaning it’s made without using human blood. […] VONVENDI is also the first and only recombinant treatment for VWD that’s approved for three uses including: Routine prophylaxis in appropriate adult patients with severe type 3 VWD, On-demand to manage active bleeds, Perioperative to help prevent bleeding episodes during or after surgery. […] In certain adult patients with severe Type 3 VWD, VONVENDI is approved to reduce the frequency of bleeding episodes. […] VONVENDI is used in adults (age 18 years and older) diagnosed with von Willebrand disease to: treat and control bleeding episodes, prevent excessive bleeding during and after surgery, reduce the number of bleeding episodes when used regularly (prophylaxis) in adults with severe Type 3 von Willebrand disease receiving on-demand therapy.

• #42 VONVENDI® Treatment for Adults With von Willebrand Disease (VWD)

  https://www.vonvendi.com/

  Your first dose of VONVENDI for each bleeding episode may be administered with a recombinant factor VIII as instructed by your healthcare provider. […] Your healthcare provider will instruct you whether additional doses of VONVENDI with or without recombinant factor VIII are needed. […] If you form inhibitors, it may stop VONVENDI or factor VIII from working properly.

• #43 VONVENDI® Prophylaxis Treatment for Von Willebrand Disease (VWD)

  https://www.vonvendipro.com/prophylaxis

  Adjust prophylaxis dose up to 60 IU/kg twice weekly if breakthrough bleeding occurs in joints or if severe bleeding occurs. Treatment of breakthrough bleeds should follow the on-demand dosing guidelines.1 […] Consider self-infusion with VONVENDI for your patients with VWD […] VONVENDI [von Willebrand factor (Recombinant)] is a recombinant von Willebrand factor (rVWF) indicated for use in adults (age 18 and older) diagnosed with von Willebrand disease (VWD) for on-demand treatment and control of bleeding episodes; perioperative management of bleeding; routine prophylaxis to reduce the frequency of bleeding episodes in patients with severe Type 3 von Willebrand disease receiving on-demand therapy.

• #44 von Willebrand Disease Treatment & Management: Approach Considerations, Type I von Willebrand disease, Type 2 von Willebrand disease

  https://emedicine.medscape.com/article/206996-treatment

  In addition, antifibrinolytic drugs (ie, aminocaproic acid, tranexamic acid) can be used orally or intravenously to treat mild mucocutaneous bleeding. […] Platelet transfusions may be helpful in some patients with vWD (eg, type 3) to control bleeding that is refractory to other therapies. […] For prophylaxis in major surgery, rVWF (with or without FVIII) or vWF-containing factor VIII (FVIII) concentrates are the treatment of choice. […] DDAVP is the treatment of choice for individuals with vWD type I. […] Responses to DDAVP are variable in patients with type 2 disease; some patients respond while others should receive vWF concentrates. […] The treatment of choice for patients with vWD type 3 (as with other vWD types unresponsive to DDAVP) is rVWF (with or without FVIII) or virus-inactivated, vWF-containing FVIII concentrates that contain a near-normal complement of high-molecular-weight vWF multimers. […] Off-label use of emicizumab, a bispecific, factor VIII-mimetic antibody approved for prophylaxis of hemophilia A, has been reported for treatment of active/recurrent bleeds in patients with type 3 vWD.

• #45 von Willebrand disease – eLearning Platform

  https://elearning.wfh.org/elearning-centres/von-willebrand-disease/

  In people with VWD who have a history of severe and frequent bleeds, it is recommended to use long-term prophylaxis with a VWF concentrate. Prophylaxis is the regular administration (intravenously, subcutaneously, or otherwise) of a hemostatic agent with the goal of preventing bleeding (especially life-threatening bleeding or recurring joint bleeding). […] As with all medications, these treatments may have side effects. People with VWD should talk to their physician about possible side effects of treatment.

• #46 Von Willebrand disease – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/von-willebrand-disease/diagnosis-treatment/drc-20354984

  Another replacement therapy approved by the Food and Drug Administration for treating adults 18 and older is a genetically engineered (recombinant) von Willebrand factor product. Because recombinant factor is made without plasma, it can reduce the risk of a viral infection or allergic reaction. […] Oral contraceptives. In addition to preventing pregnancy, these drugs can help control heavy bleeding during menstrual periods. The estrogen hormones in birth control pills can boost von Willebrand factor and factor VIII activity. […] Clot-stabilizing medications. These anti-fibrinolytic medications such as aminocaproic acid (Amicar) and tranexamic acid (Cyklokapron, Lysteda) can help stop bleeding by slowing the breakdown of blood clots. Doctors often prescribe these drugs before or after a surgical procedure or tooth extraction.

• #47 Principles of care for the diagnosis and treatment of von Willebrand disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3640108/

  Life-threatening anaphylactic reactions may occur in rare patients with type 3 VWD (typically homozygous for gene deletions or nonsense mutations) who develop alloantibodies when treated with concentrates containing VWF. […] Antifibrinolytic agents (i.e. tranexamic acid and epsilon aminocaproic acid), given orally, intravenously or topically, are useful alone or as adjuncts to replacement therapy (DDAVP or VWF/FVIII concentrates) for the prevention or treatment of bleeding in mucosal tracts, characterized by a high fibrinolytic activity. […] Estrogens-progestogen preparations may be useful to reduce the severity of menorrhagia in women with VWD, including those with type 3.

• #48 Treatment of patients with von Willebrand disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3262353/

  Von Willebrand disease (vWD) is the most common hereditary bleeding disorder. The aim of therapy is to correct the dual hemostatic defect, due to defective platelet adhesion-aggregation and abnormal coagulation due to Factor VIII (FVIII) deficiency. The choice of treatment depends on a number of factors, including the severity of the bleed, the procedure planned, the subtype and severity of the disease and the age and morbidity of the patient. Desmopressin (DDAVP) is the treatment of choice for type 1 vWD as it increases endogenous release of FVIII and von Willebrand factor (vWF) and is also used in some subtypes of type 2 vWD. In those patients in whom DDAVP is ineffective or contraindicated, levels can be restored by infusing vWF:FVIII concentrates. The role of antifibrinolytic treatment is an important adjunct to replacement therapy during minor or major surgery involving mucosal surfaces. The dosing and timing of vWF:FVIII concentrates is important depending on the nature of the surgical procedure. The role of secondary prophylaxis needs to be further defined.

• #49 Patient education: von Willebrand disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/von-willebrand-disease-beyond-the-basics

  Medications that prevent clot breakdown – Individuals with VWD may be treated with a medication that prevents blood clots from dissolving, referred to as an antifibrinolytic agent. Examples of these types of medications include aminocaproic acid (sample brand name: Amicar) and tranexamic acid (sample brand name: Cyklokapron). These medications can be given in pill form, as a mouthwash, or as an injection into a vein. […] These medications are especially useful for managing bleeding from mucosal surfaces, such as nosebleeds, heavy menstrual bleeding, or bleeding from the mouth following dental work, and they are often given in addition to other treatments for VWD. […] Treatment at the bleeding site – Some people will also benefit from application of a foam or gel to the bleeding site that stimulates clots to form right at that spot.

• #50 Diagnosis and Treatment of von Willebrand Disease and Rare Bleeding Disorders

  https://www.mdpi.com/2077-0383/6/4/45

  Additional important treatment of mucocutaneous bleeding is represented by the fibrinolysis inhibitor tranexamic acid at dosage of 15–25 mg/kg every 8 h for 3–6 days by oral or intravenous administration. In addition to systemic treatment for bleeding or surgery, the agent can be used as mouthwash for oral bleeding or dental procedures. […] To control gynaecologic bleeding, hormonal therapy is often helpful. Treatment should be initiated with oral contraceptives containing both progestin and estrogen. In those patients who do not have a significant response, a levonorgestrel-releasing intrauterine device can be used.

• #51 Exploring Therapy Options for von Willebrand Disease: What’s the Best Fit for You? – Doctronic, Your Trusted AI Doctor

  https://www.doctronic.ai/conditions-diseases/exploring-therapy-options-for-von-willebrand-disease-what-s-the-best-fit-for-you-XTs6XB

  DDAVP is typically effective for minor bleeding, especially in Type 1 VWD patients. […] For those who do not respond to DDAVP, VWF concentrates are the next option. VWF concentrates can stabilize factor VIII and are useful in more severe cases or prolonged bleeding. […] Antifibrinolytic agents like tranexamic acid help stabilize blood clots and are particularly effective for mucosal bleeds. These agents can be used alone or in conjunction with other therapies, depending on the severity and location of the bleeding. […] Topical agents like thrombin can be applied directly to bleeding areas, providing quick relief. Estrogens, often in the form of oral contraceptives, can be used to manage heavy menstrual bleeding in women with VWD. […] DDAVP increases VWF levels to manage minor bleeding.

• #52 Treatment of von Willebrand disease | Hemophilia

  https://www.hemophilia.ca/treatment-of-von-willebrand-disease/

  In major surgery, desmopressin alone may not be enough to control bleeding. In such a case, a person should also receive a concentrate of VWF and factor VIII. […] Desmopressin can sometimes have some mild side effects. These are: facial flushing, mild headache, nausea and abdominal cramps. […] Cyklokapron (tranexamic acid) and Amicar (aminocaproic acid) are drugs that help to hold a clot in place once it has formed. […] They can be used to hold a clot in place in mucous membranes such as: the inside of the mouth, the inside of the nose, inside the intestines (the gut), inside the uterus (the womb). […] Factor VIII/von Willebrand factor concentrate (FVIII/VWF concentrate) is the preferred treatment for: Type 3 VWD, most forms of Type 2 VWD, for serious bleeding or major surgery in all types of VWD. […] FVIII/VWF concentrate is injected into a vein. It can be administered at a clinic, doctor’s office or emergency room. Many people learn to inject it themselves at home.

• #53 Treatment of von Willebrand Disorder – Irish Haemophilia Society

  https://haemophilia.ie/about-bleeding-disorders/vwd-2/treatment/

  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.

• #54 Treatment of Von Willebrand Disease | Hematology-Oncology Associates of CNY

  https://www.hoacny.com/patient-resources/blood-disorders/what-von-willebrand-disease/treatment-von-willebrand-disease

  Antifibrinolytic (AN-te-fi-BRIN-o-LIT-ik) medicines also are used to treat VWD. These medicines help prevent the breakdown of blood clots. They’re mostly used to stop bleeding after minor surgery, tooth extraction, or an injury. These medicines may be used alone or with desmopressin and replacement therapy. […] Treatments for women who have VWD with heavy menstrual bleeding include: Birth control pills. The hormones in these pills can increase the amount of von Willebrand factor and factor VIII in your blood. The hormones also can reduce menstrual blood loss. Birth control pills are the most recommended birth control method for women who have VWD. […] For some women who are done having children or don’t want children, endometrial ablation (EN-do-ME-tre-al ab-LA-shun) is done. This procedure destroys the lining of the uterus. It has been shown to reduce menstrual blood loss in women who have VWD. […] If you need a hysterectomy (HIS-ter-EK-to-me; surgical removal of the uterus) for another reason, this procedure will stop menstrual bleeding and possibly improve your quality of life. However, hysterectomy has its own risk of bleeding complications.

• #55 Von Willebrand Disease | Boston Children’s Hospital

  https://www.childrenshospital.org/conditions/von-willebrand-disease

  Von Willebrand Factor replacement therapies: Special concentrate of VWF (as well as factor VIII) are available for treatment of VWD. The VWF concentrates are administered by infusion (injections into a vein) and allow for replacement of VWF. This treatment is used if your child does not respond to DDAVP, needs therapy for an extended time, or has a severe injury. […] Antifibrinolytic agents: Oral or IV medicines such as aminocaproic acid and tranexamic acid can be used to help make clots more stable. These medications may be particularly helpful for managing bloody noses (epistaxis), heavy menstrual bleeding, and after dental procedures for patients with bleeding disorders. An antifibrinolytic agent may be used alone or combined with DDAVP or VWF replacement therapy. […] Your VWD treatment team will take many considerations into account to ensure the best treatment and follow-up plan for your child including: Your child’s age, overall health and medical history, The severity of the disease, Your child’s tolerance for certain medications, procedures or therapies, How your child’s doctors expect the disease to progress, Your opinion and preferences.

• #56 Treatment of von Willebrand Disorder – Irish Haemophilia Society

  https://haemophilia.ie/about-bleeding-disorders/vwd-2/treatment/

  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.

• #57 Von Willebrand disease – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/von-willebrand-disease/diagnosis-treatment/drc-20354984

  Another replacement therapy approved by the Food and Drug Administration for treating adults 18 and older is a genetically engineered (recombinant) von Willebrand factor product. Because recombinant factor is made without plasma, it can reduce the risk of a viral infection or allergic reaction. […] Oral contraceptives. In addition to preventing pregnancy, these drugs can help control heavy bleeding during menstrual periods. The estrogen hormones in birth control pills can boost von Willebrand factor and factor VIII activity. […] Clot-stabilizing medications. These anti-fibrinolytic medications such as aminocaproic acid (Amicar) and tranexamic acid (Cyklokapron, Lysteda) can help stop bleeding by slowing the breakdown of blood clots. Doctors often prescribe these drugs before or after a surgical procedure or tooth extraction.

• #58 Patient education: von Willebrand disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/von-willebrand-disease-beyond-the-basics

  Treatment for control of menstrual bleeding – Your doctor or nurse may refer you to a gynecologist who can determine whether the bleeding is caused by VWD or by something gynecologic such as a polyp. […] People with VWD who have heavy periods are sometimes treated with hormones, such as birth control pills or a progestin-releasing intrauterine device (IUD). […] Some people also benefit from DDAVP or an antifibrinolytic medicine that prevents the breakdown of clots (for example, tranexamic acid; sample brand name Cyklokapron). These treatments can reduce heavy menstrual bleeding. In some patients for whom these measures are not effective, a VWF concentrate is given. […] Treatment during pregnancy and childbirth – Many people with VWD who become pregnant can have a normal pregnancy without bleeding complications.

• #59 Medications for von Willebrand Disease | NYU Langone Health

  https://nyulangone.org/conditions/von-willebrand-disease-in-children/treatments/medications-for-von-willebrand-disease

  Contraceptives that contain hormones can help to limit blood loss during menstrual periods. Our doctors may recommend that adolescent girls with von Willebrand disease who have heavy menstrual periods take oral contraceptives, or birth control pills. […] For young adult women, our doctors may recommend a hormone-releasing intrauterine device, also known as an IUD. Some teens and women with von Willebrand disease require a combination of hormonal contraceptives with antifibrinolytics or DDAVP to treat heavy menstrual flow.

• #60 Treatment of Von Willebrand Disease | Hematology-Oncology Associates of CNY

  https://www.hoacny.com/patient-resources/blood-disorders/what-von-willebrand-disease/treatment-von-willebrand-disease

  Antifibrinolytic (AN-te-fi-BRIN-o-LIT-ik) medicines also are used to treat VWD. These medicines help prevent the breakdown of blood clots. They’re mostly used to stop bleeding after minor surgery, tooth extraction, or an injury. These medicines may be used alone or with desmopressin and replacement therapy. […] Treatments for women who have VWD with heavy menstrual bleeding include: Birth control pills. The hormones in these pills can increase the amount of von Willebrand factor and factor VIII in your blood. The hormones also can reduce menstrual blood loss. Birth control pills are the most recommended birth control method for women who have VWD. […] For some women who are done having children or don’t want children, endometrial ablation (EN-do-ME-tre-al ab-LA-shun) is done. This procedure destroys the lining of the uterus. It has been shown to reduce menstrual blood loss in women who have VWD. […] If you need a hysterectomy (HIS-ter-EK-to-me; surgical removal of the uterus) for another reason, this procedure will stop menstrual bleeding and possibly improve your quality of life. However, hysterectomy has its own risk of bleeding complications.

• #61 Diagnosis and Treatment of von Willebrand Disease and Rare Bleeding Disorders

  https://www.mdpi.com/2077-0383/6/4/45

  Additional important treatment of mucocutaneous bleeding is represented by the fibrinolysis inhibitor tranexamic acid at dosage of 15–25 mg/kg every 8 h for 3–6 days by oral or intravenous administration. In addition to systemic treatment for bleeding or surgery, the agent can be used as mouthwash for oral bleeding or dental procedures. […] To control gynaecologic bleeding, hormonal therapy is often helpful. Treatment should be initiated with oral contraceptives containing both progestin and estrogen. In those patients who do not have a significant response, a levonorgestrel-releasing intrauterine device can be used.

• #62 Von Willebrand Disease in Women | ACOG

  https://www.acog.org/clinical/clinical-guidance/committee-opinion/articles/2013/12/von-willebrand-disease-in-women

  Studies in women with von Willebrand disease and heavy menstrual bleeding suggest that the levonorgestrel-releasing intrauterine system may be effective for this population. […] Nonhormonal treatment options include antifibrinolytic agents, such as tranexamic acid and -aminocaproic acid, and treatments that increase endogenous plasma concentration of vWF, replace vWF, or promote hemostasis without affecting vWF. […] Therapies generally prescribed in conjunction with a hematologist once a diagnosis of von Willebrand disease is established include desmopressin acetate, recombinant factor VIII, and vWF complex infusion. […] Once a diagnosis of von Willebrand disease has been established, a multidisciplinary approach to management, which involves obstetriciangynecologists and hematologists, results in optimal treatment outcomes.

• #63 Treatment of Von Willebrand Disease | Hematology-Oncology Associates of CNY

  https://www.hoacny.com/patient-resources/blood-disorders/what-von-willebrand-disease/treatment-von-willebrand-disease

  Antifibrinolytic (AN-te-fi-BRIN-o-LIT-ik) medicines also are used to treat VWD. These medicines help prevent the breakdown of blood clots. They’re mostly used to stop bleeding after minor surgery, tooth extraction, or an injury. These medicines may be used alone or with desmopressin and replacement therapy. […] Treatments for women who have VWD with heavy menstrual bleeding include: Birth control pills. The hormones in these pills can increase the amount of von Willebrand factor and factor VIII in your blood. The hormones also can reduce menstrual blood loss. Birth control pills are the most recommended birth control method for women who have VWD. […] For some women who are done having children or don’t want children, endometrial ablation (EN-do-ME-tre-al ab-LA-shun) is done. This procedure destroys the lining of the uterus. It has been shown to reduce menstrual blood loss in women who have VWD. […] If you need a hysterectomy (HIS-ter-EK-to-me; surgical removal of the uterus) for another reason, this procedure will stop menstrual bleeding and possibly improve your quality of life. However, hysterectomy has its own risk of bleeding complications.

• #64 Patient education: von Willebrand disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/von-willebrand-disease-beyond-the-basics

  Treatment for control of menstrual bleeding – Your doctor or nurse may refer you to a gynecologist who can determine whether the bleeding is caused by VWD or by something gynecologic such as a polyp. […] People with VWD who have heavy periods are sometimes treated with hormones, such as birth control pills or a progestin-releasing intrauterine device (IUD). […] Some people also benefit from DDAVP or an antifibrinolytic medicine that prevents the breakdown of clots (for example, tranexamic acid; sample brand name Cyklokapron). These treatments can reduce heavy menstrual bleeding. In some patients for whom these measures are not effective, a VWF concentrate is given. […] Treatment during pregnancy and childbirth – Many people with VWD who become pregnant can have a normal pregnancy without bleeding complications.

• #65 Von Willebrand Disease: Symptoms, Causes, Diagnosis, Treatment

  https://www.webmd.com/a-to-z-guides/von-willebrand-disease-vwd

  Your doctor might also recommend that you get clotting factor concentrates through an IV. […] If youre going to have a dental procedure, you may need to take aminocaproic acid or tranexamic acid. These prevent the breakdown of blood clots. You take these medications by mouth, either in liquid or pill form. You may also take them if you’re bleeding from your nose or mouth, or if you have heavy bleeding with your period. […] If youre a woman with VWD and heavy periods, your treatment may also include birth control pills. These can increase the amount of von Willebrand factor in your blood. Another possible treatment is a levonorgestrel intrauterine device. This is a type of birth control that contains the hormone progestin. If youre done having children or dont want any, you can also have an endometrial ablation. This procedure destroys the lining of the uterus and reduces the amount of blood you lose during your period. […] If you have Type 3 and are experiencing bleeding, you need to get treatment right away. Bleeding episodes can be fatal if not treated immediately.

• #66 Patient education: von Willebrand disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/von-willebrand-disease-beyond-the-basics

  Treatment for control of menstrual bleeding – Your doctor or nurse may refer you to a gynecologist who can determine whether the bleeding is caused by VWD or by something gynecologic such as a polyp. […] People with VWD who have heavy periods are sometimes treated with hormones, such as birth control pills or a progestin-releasing intrauterine device (IUD). […] Some people also benefit from DDAVP or an antifibrinolytic medicine that prevents the breakdown of clots (for example, tranexamic acid; sample brand name Cyklokapron). These treatments can reduce heavy menstrual bleeding. In some patients for whom these measures are not effective, a VWF concentrate is given. […] Treatment during pregnancy and childbirth – Many people with VWD who become pregnant can have a normal pregnancy without bleeding complications.

• #67 Patient education: von Willebrand disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/von-willebrand-disease-beyond-the-basics

  However, as with surgery, it is important to have proper monitoring and to know what treatments will work well for you if you do have excessive bleeding. If you get pregnant or want to try to get pregnant, you should discuss your VWD with your hematologist. A specialist with expertise in managing pregnancy in people with VWD should be involved, and close monitoring during the pregnancy is appropriate. […] These discussions will help your health care team develop a plan to control any excessive bleeding before it becomes a problem. […] Many people will not need treatment while they are pregnant because VWF levels naturally increase during pregnancy. After delivery, however, levels of VWF can fall quickly and lead to more prolonged or heavier bleeding. One or more of the treatments described above may be used for one to three weeks following delivery.

• #68 Diagnosis and Management of Von Willebrand Disease: Guidelines for Primary Care | AAFP

  https://www.aafp.org/pubs/afp/issues/2009/1201/p1261.html

  Desmopressin is a synthetic derivative of the antidiuretic hormone. It is used in type 1 VWD and in some patients with type 2 VWD. […] Humate-P and Alphanate are currently the only plasma-derived VWF concentrates approved by the U.S. Food and Drug Administration for the treatment of VWD. […] The oral antifibrinolytic agents epsilon-aminocaproic acid (Amicar) and tranexamic acid (oral formulation no longer available in the United States) are very useful for mucous membrane bleeding, such as with dental procedures. […] If menorrhagia is caused by VWD and the patient is not trying to get pregnant, combined oral contraceptives are the treatment of choice. The levonorgestrel-releasing intrauterine device (Mirena) is an alternative in appropriate patients. […] Desmopressin therapy initiated during labor may cause significant fluid retention, which is aggravated by use of oxytocin (Pitocin), with risk of hyponatremia and seizures. Therefore, women with VWD and factor VIII levels or VWF:RCo of less than 50 IU per dL should receive VWF replacement with VWF concentrates as opposed to desmopressin.

• #69 Patient education: von Willebrand disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/von-willebrand-disease-beyond-the-basics

  However, as with surgery, it is important to have proper monitoring and to know what treatments will work well for you if you do have excessive bleeding. If you get pregnant or want to try to get pregnant, you should discuss your VWD with your hematologist. A specialist with expertise in managing pregnancy in people with VWD should be involved, and close monitoring during the pregnancy is appropriate. […] These discussions will help your health care team develop a plan to control any excessive bleeding before it becomes a problem. […] Many people will not need treatment while they are pregnant because VWF levels naturally increase during pregnancy. After delivery, however, levels of VWF can fall quickly and lead to more prolonged or heavier bleeding. One or more of the treatments described above may be used for one to three weeks following delivery.

• #70 Von Willebrand Disease | Condition | UT Southwestern Medical Center

  https://utswmed.org/conditions-treatments/von-willebrand-disease/

  People with mild cases may need treatment only after an injury or before surgery or dental work. […] People with von Willebrand disease who are considering becoming pregnant might want to speak with their doctor about how to reduce risk of complications. […] In addition to treatment, it may be helpful to: […] Discuss pain relief options with a doctor; it might make sense to take acetaminophen instead of blood-thinning drugs such as aspirin, ibuprofen, and naproxen sodium […] Alert all care providers, including doctors and dentists, about a diagnosis or family history of von Willebrand disease […] Wear a medical ID bracelet and carry a medical alert card […] Exercise (while avoiding contact sports).

• #71 Diagnosis and Management of Von Willebrand Disease: Guidelines for Primary Care | AAFP

  https://www.aafp.org/pubs/afp/issues/2009/1201/p1261.html

  Desmopressin is a synthetic derivative of the antidiuretic hormone. It is used in type 1 VWD and in some patients with type 2 VWD. […] Humate-P and Alphanate are currently the only plasma-derived VWF concentrates approved by the U.S. Food and Drug Administration for the treatment of VWD. […] The oral antifibrinolytic agents epsilon-aminocaproic acid (Amicar) and tranexamic acid (oral formulation no longer available in the United States) are very useful for mucous membrane bleeding, such as with dental procedures. […] If menorrhagia is caused by VWD and the patient is not trying to get pregnant, combined oral contraceptives are the treatment of choice. The levonorgestrel-releasing intrauterine device (Mirena) is an alternative in appropriate patients. […] Desmopressin therapy initiated during labor may cause significant fluid retention, which is aggravated by use of oxytocin (Pitocin), with risk of hyponatremia and seizures. Therefore, women with VWD and factor VIII levels or VWF:RCo of less than 50 IU per dL should receive VWF replacement with VWF concentrates as opposed to desmopressin.

• #72 Principles of care for the diagnosis and treatment of von Willebrand disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC3640108/

  The first step is to carry out a test with desmopressin. Candidates are those patients with basal FVIII and/or VWF:RCo below 30 U/dL. […] DDAVP increases VWF and FVIII plasma concentrations in patients with mild hemophilia A and VWD by provoking release of stored VWF. […] Those patients in whom a test infusion with desmopressin is not able to achieve clinically useful FVIII and/or VWF levels are candidates for replacement therapy. […] VWF and FVIII levels in VWD can be normalized by the infusion of virally-inactivated plasma-derived concentrates containing both these proteins and these concentrates are the mainstay of treatment for patients not candidates for the use of DDAVP. […] Some patients with severe forms of VWD (FVIII:C levels 5 U/dL) may have frequent hemarthroses or recurrent spontaneous bleeding (e.g. gastrointestinal bleeding and epistaxis) which can benefit from secondary long-term prophylaxis.

• #73 Von Willebrand Disease Treatment Guidelines | NBDF

  https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-266-masac-recommendations-regarding-the-treatment-of-von-willebrand-disease

  3. VWF Replacement […] Persons with type 2B and type 3 VWD, and those with type 1, 2A, 2M, and 2N who have been shown to be nonresponsive to DDAVP, should be treated with a factor VIII/VWF concentrate that is known to contain the higher molecular weight multimers of von Willebrand factor and that has been virally attenuated to eliminate transmission of HIV and hepatitis A, B, and C. Human plasma-derived products Alphanate, Humate P, and Wilate have been approved by the FDA for use in VWD. A recombinant VWF concentrate, Vonvendi, has also been approved. […] […] 4. Antifibrinolytics […] Adjunctive treatments for mucous membrane bleeding include the antifibrinolytic agents aminocaproic acid and tranexamic acid. These agents can be given orally or intravenously. […] […] 6. Prophylaxis […] In patients with VWD with a history of major and frequent bleeds, the ASH ISTH NHF WFH guideline panel suggests using long-term prophylaxis with factor replacement rather than no prophylaxis.(3) Prophylaxis in VWD is defined as a period of at least 3 months of treatment of VWF concentrate at least once weekly, or for women with HMB, use of VWF concentrate at least once per menstrual cycle. […]

• #74 Patient education: von Willebrand disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/von-willebrand-disease-beyond-the-basics

  Treatment of severe bleeding – If you are in an accident or have severe bleeding with surgery despite receiving the treatments above, you may be given additional VWF, other medications, and/or platelet transfusions. It may be necessary to receive treatment for several days when severe bleeding occurs. […] It is important to carry information about your VWD, including what type of VWD you have (see 'Types of von Willebrand disease’ above) and a list of any other medical problems and medications on a medical identification card or bracelet. This will help your health care providers know what type of treatment is likely to work best for you. […] Treatment of recurrent bleeding – Some individuals with severe VWD who have repeated, frequent bleeding in an area such as in a joint or from the gastrointestinal tract may benefit from intravenous injections of a VWF concentrate on a regular basis, often two to three times weekly. This is referred to as „prophylaxis.”

• #75 Diagnosis and Treatment of von Willebrand Disease and Rare Bleeding Disorders

  https://www.mdpi.com/2077-0383/6/4/45

  For those VWD patients in whom DDAVP is either ineffective or contraindicated, replacement therapy with pd-VWF/FVIII concentrates is the treatment of choice. Current effective intermediate and high-purity VWF-FVIII concentrates are several and all plasma-derived, with a variable content of VWF and FVIII, as well as a heterogeneous multimer pattern. […] Different recommendations or national guidelines for VWD treatment are available, and useful indications for replacement therapy in spontaneous bleedings, minor and major surgeries, delivery and puerperium, development of alloantibodies are summarized. […] In contrast to patients with haemophilia, regular replacement therapy to prevent bleeding is not commonly used in those with VWD, even if its benefit has been described in several case series. In patients with severe forms of VWD, when plasma FVIII level is <5 U/dL, suffering from recurrent joint bleeding or gastrointestinal bleeding, a secondary long-term prophylaxis may represent an efficacious treatment.

• #76 WIL-31: Expanding treatment options for VWD

  https://www.octapharma.com/news/innovation-and-science/2024/wil-31-expanding-treatment-options-for-vwd

  „The approval of Octapharma’s von Willebrand factor for VWD prophylaxis could improve the quality of life for many patients and I’m proud to have led the team that achieved that.” […] „On December 1, 2023 the US Food Drug Administration (FDA) granted expanded approval to Octapharma’s von Willebrand factor (VWF), making it the first von Willebrand factor concentrate indicated for prophylactic treatment across all forms of von Willebrand disease (VWD).” […] „The label expansion thus offers an invaluable new treatment option for a significant number of patients.” […] „Long-term prophylaxis with VWF concentrate, as compared with on-demand treatment for bleeding, is recommended for patients with severe VWD.” […] „The findings from WIL-31 provide strong evidence to support the use of VWF prophylaxis and have led to the recent addition of wilate prophylaxis as a therapeutic indication for VWD in the USA, widening its access to the US patient population.”

• #77 Breaking new ground in von Willebrand disease treatment

  https://www.labiotech.eu/trends-news/von-willebrand-disease-treatment-advancements/

  More tangible perhaps, in December 2023, the FDA granted expanded approval to Octapharmas VWF concentrate, making it the first VWF concentrate indicated for prophylactic treatment across all forms of VWD. […] As new treatments advance to reach the clinic the burden on von Willebrand disease patients could become lighter in the years to come.

• #78 Improving Treatment for Women and Girls With von Willebrand Disease – Hematology Advisor

  https://www.hematologyadvisor.com/features/von-willebrand-disease-vwd-improve-treatment-women-girls-treatment-risk/

  Researchers sought to highlight treatments that improve quality of life in girls, women, and people with the potential to menstruate with von Willebrand disease. Emerging data on emicizumab, BT200 (rondoraptivon pegol), generalized hemostatic therapies (VGA039 and HMB-011), and treatments based on nanotechnology have demonstrated great promise for girls, women, and people with the potential to menstruate with von Willebrand disease. […] The authors noted that innovation in therapies for patients with VWD has lagged far behind that for hemophilia, creating inequity in the bleeding disorder community. They wrote in their report that current treatments include antifibrinolytics, desmopressin, and plasma-derived von Willebrand factor (VWF) replacement. […] Recombinant VWF is the most recent therapy to become available for patients with VWD. Its pharmacokinetics, safety, and efficacy have been demonstrated in male and female patients.

• #79 Improving Treatment for Women and Girls With von Willebrand Disease – Hematology Advisor

  https://www.hematologyadvisor.com/features/von-willebrand-disease-vwd-improve-treatment-women-girls-treatment-risk/

  Emicizumab is a bispecific monoclonal antibody that mimics the function of FVIII by bridging activated factor IX and factor X. […] BT200 (rondoraptivon pegol) is a pegylated aptamer that binds to the VWF A1 domain. […] VGA039 is a monoclonal antibody directed against human protein S that inhibits its cofactor activity for tissue factor pathway inhibitor and activated protein C, enhancing thrombin generation through both the initiation and propagation phases of coagulation. […] HMB-001 is a bispecific antibody that binds and accumulates endogenous activated FVII in circulation. […] Synthetic platelet nanoparticles are small liposomal particles coated with multiple peptides facilitating concomitant binding to collagen. […] A bifunctional molecule that binds both albumin and VWF, KB-V13A12 prolongs the half-life of endogenous VWF, but it is still in its early stages of development.

• #80 An overview of novel innovative treatments for patients with von Willebrand disease | VJHemOnc

  https://www.vjhemonc.com/video/gteuz7mge5s-an-overview-of-novel-innovative-treatments-for-patients-with-von-willebrand-disease/

  First, some therapies aim to increase endogenous von Willebrand factor (vWF) levels, such as an albumin- and vWF-targeted bispecific nanobody for patients with vWD type 1, or BT200, a vWF-targeted aptamer, for those with type 2b disease. […] Second, treatments that increase procoagulant potential, such as emicizumab or BIVV001, could benefit specific subsets of patients. […] Thirdly, general hemostatic agents such as platelet inspired nanoparticles could help to reduce bleeding symptoms. […] Dr Casari then goes on to discuss the challenges of using gene therapies in this disease, as well as the potential of silencing RNA (siRNA) approaches.

• #81 Improving Treatment for Women and Girls With von Willebrand Disease – Hematology Advisor

  https://www.hematologyadvisor.com/features/von-willebrand-disease-vwd-improve-treatment-women-girls-treatment-risk/

  Emicizumab is a bispecific monoclonal antibody that mimics the function of FVIII by bridging activated factor IX and factor X. […] BT200 (rondoraptivon pegol) is a pegylated aptamer that binds to the VWF A1 domain. […] VGA039 is a monoclonal antibody directed against human protein S that inhibits its cofactor activity for tissue factor pathway inhibitor and activated protein C, enhancing thrombin generation through both the initiation and propagation phases of coagulation. […] HMB-001 is a bispecific antibody that binds and accumulates endogenous activated FVII in circulation. […] Synthetic platelet nanoparticles are small liposomal particles coated with multiple peptides facilitating concomitant binding to collagen. […] A bifunctional molecule that binds both albumin and VWF, KB-V13A12 prolongs the half-life of endogenous VWF, but it is still in its early stages of development.

• #82 Improving Treatment for Women and Girls With von Willebrand Disease – Hematology Advisor

  https://www.hematologyadvisor.com/features/von-willebrand-disease-vwd-improve-treatment-women-girls-treatment-risk/

  Current research includes investigation into the potential efficacy of emicizumab for patients with VWD who clinically require prophylactic therapy after demonstration of efficacy in small case studies. […] Finally, VEGA therapeutics is conducting a phase 1 trial of their VGA039, anti-protein S, monoclonal antibody (ClincialTrials.gov Identifier: NCT05776069). VGA039 is the only molecule in phase 1 that was designed to specifically focus on treatment of VWD, although I think we are a while away from commercial availability, said Dr Wheeler.

• #83 Breaking new ground in von Willebrand disease treatment

  https://www.labiotech.eu/trends-news/von-willebrand-disease-treatment-advancements/

  Antifibrinolytic agents such as tranexamic and aminocaproic acids are used as adjunctive therapies to prevent the breakdown of blood clots. […] A notable advantage of VGA039 is its subcutaneous administration, which contrasts with the intravenous infusions required by current von Willebrand disease prophylaxis therapies. […] Interim clinical data from the VIVID 2 study presented at the American Society of Hematology (ASH) 2024 meeting demonstrated that a single subcutaneous dose of VGA039 sustained multi-week therapeutic concentrations. […] Emicizumab is a bispecific monoclonal antibody that mimics the function of factor VIII by bridging activated factor IX and factor X, facilitating effective clot formation. […] Band Therapeutics BT200 is currently in multiple clinical trials for different subtypes of von Willebrand disease it is most advanced for type 2B where its in phase 3 and its in phase 2 for type 1 and 3.

• #84 Breaking new ground in von Willebrand disease treatment

  https://www.labiotech.eu/trends-news/von-willebrand-disease-treatment-advancements/

  Earlier this week, Star Therapeutics von Willebrand disease candidate VGA039 was granted Fast Track designation by the U.S. Food and Drug Administration (FDA). […] The inherited disease is managed with different strategies depending on the type and severity of the disorder. The primary goal remains controlling bleeding episodes. […] Desmopressin is one of the standard solutions for von Willebrand disease. It is a synthetic hormone that stimulates the release of stored VWF, thereby increasing their levels in the bloodstream. […] For patients who do not respond to desmopressin or require more attention because of more severe bleeding, VWF replacement strategies are used. These include plasma-derived concentrates, derived from human plasma, and contain both VWF and factor VIII. […] Recombinant VWF is another approach with Vonvendi being the first and only recombinant treatment approved for von Willebrand disease. This is used as an on-demand control treatment of bleeding episodes as well as a routine treatment to reduce the frequency of these episodes.

• #85 Breaking new ground in von Willebrand disease treatment

  https://www.labiotech.eu/trends-news/von-willebrand-disease-treatment-advancements/

  Antifibrinolytic agents such as tranexamic and aminocaproic acids are used as adjunctive therapies to prevent the breakdown of blood clots. […] A notable advantage of VGA039 is its subcutaneous administration, which contrasts with the intravenous infusions required by current von Willebrand disease prophylaxis therapies. […] Interim clinical data from the VIVID 2 study presented at the American Society of Hematology (ASH) 2024 meeting demonstrated that a single subcutaneous dose of VGA039 sustained multi-week therapeutic concentrations. […] Emicizumab is a bispecific monoclonal antibody that mimics the function of factor VIII by bridging activated factor IX and factor X, facilitating effective clot formation. […] Band Therapeutics BT200 is currently in multiple clinical trials for different subtypes of von Willebrand disease it is most advanced for type 2B where its in phase 3 and its in phase 2 for type 1 and 3.

• #86 Patient education: von Willebrand disease (Beyond the Basics) – UpToDate

  https://www.uptodate.com/contents/von-willebrand-disease-beyond-the-basics

  VON WILLEBRAND DISEASE TREATMENT […] If you are diagnosed with von Willebrand disease (VWD), you should be referred to a hematologist. They usually recommend that you avoid medications that interfere with the function of your platelets. These might include aspirin and medications known as nonsteroidal anti-inflammatory drugs (NSAIDs), such as ibuprofen (sample brand names: Advil, Motrin) and naproxen (sample brand name: Aleve). If you need a medication for pain or fever relief, acetaminophen (sample brand name: Tylenol) is a good alternative to aspirin and NSAIDs. If you take one of these medicines for another reason such as heart disease or stroke, do not stop them until you speak with the doctor who prescribed them. […] Depending on the severity of your VWD, you may also have to take special precautions to avoid injuries, such as avoiding contact sports. However, such precautions may not be necessary for most people with mild type 1 VWD.

• #87 Treatment for von Willebrand Disease > Von Willebrand Disease > Bleeding Disorders > HoG Handbook > Hemophilia of Georgia

  https://www.hog.org/handbook/article/1/7/treatment-for-von-willebrand-disease

  In general, people with mild to moderate VWD can expect few problems with bleeding except when having surgery. A person with severe VWD may have many of the same problems that a person with Hemophilia has, including bleeding into joints. They need to treat bleeds early, visit an HTC regularly, and be under the care of a hematologist. People with VWD should not take aspirin or ibuprofen or medicines that contain them. The aspirin will make the platelets not work as well. It is best to ask the pharmacist before using any drug off the shelf. Aspirin is put in some of the least-likely medicines. Taking large amounts of vitamin E can also mess up your platelets. They cant stick together as well. […] People with VWD should carry medical alert information like wallet cards, necklaces, and bracelets. These should include their disease, their factor levels, and how they are treated for bleeding.

• #88 Von Willebrand disease: Medications to avoid

  https://www.medicalnewstoday.com/articles/medications-to-avoid-with-von-willebrand-disease

  Because von Willebrand disease (VWD) is a bleeding disorder, people with the condition should avoid medications that can increase the risk of bleeding or interfere with blood clotting. […] The following medications and therapies can help treat VWD and related symptoms: Desmopressin (DDAVP): This synthetic form of the natural hormone, vasopressin, promotes the release of von Willebrand factor and factor VIII into the bloodstream. It is available as an injectable or inhalant and is a common treatment for mild bleeds. […] Factor replacement therapies: These therapies elevate the concentration of factors in the body, promoting improved clotting capabilities. […] Antifibrinolytic drugs: These are clot-stabilizing medications that help slow or stop the breakdown of blood clots and are especially useful in the perioperative management of people with VWD.

• #89 Von Willebrand Disease | Boston Children’s Hospital

  https://www.childrenshospital.org/conditions/von-willebrand-disease

  Von Willebrand Disease is a lifelong condition with no cure. The long-term outlook for children with VWD is generally good and most have a normal lifespan. Throughout their lifetime, many patients require periodic treatment for bleeding symptoms or treatment before surgery or dental procedures. We generally recommend avoiding aspirin and ibuprofen (NSAIDS), as well as products containing them, since they may increase bleeding symptoms in children with bleeding disorders. […] We treat our patients who have von Willebrand Disease within the Boston Bleeding Disorders Center, a federally supported joint program between Boston Children’s Hospital and Brigham and Women’s Physicians Organization. The Boston Bleeding Disorders Center is the largest hemophilia treatment program in New England, and it provides treatment for a variety of bleeding disorders, including VWD.

• #90 Von Willebrand Disease Information & Treatment

  https://www.columbiadoctors.org/health-library/condition/von-willebrand-disease/

  Treatment depends on the type of von Willebrand disease you have, how much you bleed, and your risk for heavy bleeding. […] If you have von Willebrand disease, your treatment may include: […] Desmopressin medicine (such as DDAVP, Stimate) which helps your body release more of the von Willebrand clotting factor into your blood. […] Replacement therapy, which replaces the clotting factor in your blood. […] Medicines to help stop blood clots from breaking down. […] Birth control pills, or an intrauterine device (IUD) that contains hormones, to help control heavy menstrual periods. […] If you have von Willebrand disease, you most likely will need to take extra care to treat and prevent bleeding episodes. Avoid NSAIDs and blood thinners. Try to stay at a healthy weight and keep active. This helps keep your muscles and joints healthy. Avoid sports or activities where injury and bleeding are likely, such as football and hockey.

• #91 Von Willebrand Disease | Condition | UT Southwestern Medical Center

  https://utswmed.org/conditions-treatments/von-willebrand-disease/

  People with mild cases may need treatment only after an injury or before surgery or dental work. […] People with von Willebrand disease who are considering becoming pregnant might want to speak with their doctor about how to reduce risk of complications. […] In addition to treatment, it may be helpful to: […] Discuss pain relief options with a doctor; it might make sense to take acetaminophen instead of blood-thinning drugs such as aspirin, ibuprofen, and naproxen sodium […] Alert all care providers, including doctors and dentists, about a diagnosis or family history of von Willebrand disease […] Wear a medical ID bracelet and carry a medical alert card […] Exercise (while avoiding contact sports).

• #92 Von Willebrand Disease | Boston Children’s Hospital

  https://www.childrenshospital.org/conditions/von-willebrand-disease

  Von Willebrand Disease is a lifelong condition with no cure. The long-term outlook for children with VWD is generally good and most have a normal lifespan. Throughout their lifetime, many patients require periodic treatment for bleeding symptoms or treatment before surgery or dental procedures. We generally recommend avoiding aspirin and ibuprofen (NSAIDS), as well as products containing them, since they may increase bleeding symptoms in children with bleeding disorders. […] We treat our patients who have von Willebrand Disease within the Boston Bleeding Disorders Center, a federally supported joint program between Boston Children’s Hospital and Brigham and Women’s Physicians Organization. The Boston Bleeding Disorders Center is the largest hemophilia treatment program in New England, and it provides treatment for a variety of bleeding disorders, including VWD.

• #93 Von Willebrand Disease Treatment | St. Jude Care & Treatment

  https://www.stjude.org/care-treatment/treatment/blood-disorders/von-willebrand-disease.html

  Treatment for von Willebrand disease is based on the type and severity of the disorder. If your child has type 1 von Willebrand disease, treatment may only be needed if they have surgery, a dental procedure, or a traumatic injury. If your child has more severe von Willebrand disease, they may need regular treatments to prevent or stop life-threatening bleeds. […] Current therapies for von Willebrand disease include: Desmopressin acetate (DDVAP) is a medicine that stimulates the release of more von Willebrand factor into the blood. Von Willebrand factor replacement therapy may be used to replace the clotting factor that is low or missing in the blood. Other medicines such as antifibrinolytic medicines and fibrin glue may be used to help the blood to clot. […] With proper medical care, most children with von Willebrand disease can live a normal, active life.

• #94 Von Willebrand disease – Symptoms & causes – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/von-willebrand-disease/symptoms-causes/syc-20354978

  Von Willebrand disease can’t be cured. But with treatment and self-care, most people with this disease can lead active lives. […] Rick ME. Von Willebrand disease: Treatment of minor bleeding and routine care. […] Rick ME. Von Willebrand disease: Treatment of major bleeding and major surgery. […] Connell NT, et al. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease.

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