Materiały źródłowe

• #1 Prophylactic management of patients with von Willebrand disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8721401/

  Von Willebrand disease, the most common inherited bleeding disorder that affects both males and females, is due to quantitative or qualitative defects of the multimeric glycoprotein von Willebrand factor, which cause mucous membrane bleeding but also soft tissue bleeding owing to the secondary deficiency of factor VIII. The aim of treatment is to correct this dual defect of hemostasis. In addition to the episodic management of bleeding episodes, therapy includes their short- or long-term prevention. Short-term prophylaxis is mainly warranted in order to provide effective hemostatic coverage to patients undergoing surgery or invasive procedures and to affected women at the time of delivery or during menstruations associated with excessive bleeding. The aim of long-term prophylaxis is to prevent bleeding in particular categories of patients at increased risk of frequent and spontaneous bleeding in the joints, nose, and gastrointestinal tract.

• #1 Prophylaxis for Bleeding Episodes in Severe Type 3 von Willebrand Disease  – Hematology Advisor

  https://www.hematologyadvisor.com/cch/type-3-vwd-prophylaxis-rvwf-pdvwf-bleeding-clotting/

  Prophylaxis for Bleeding Episodes in Severe Type 3 von Willebrand Disease […] The on-demand treatment protocol for VWD is an established management strategy, which focuses on the episodic correction of the hemostatic defects during bleeding episodes. […] However, it has been speculated that people with type 3 VWD can benefit from long-term prophylaxis, which is expected to lower annual bleeding events. […] Short-term prophylaxis is also indicated in pregnancy for patients with type 3 VWD. […] Therefore, prenatal care and delivery should be performed in centers that, in addition to specialists in high-risk obstetrics, have a hemophilia treatment center and/or a hematologist with expertise in hemostasis, and prophylaxis should be offered to these patients before invasive procedures such as chorionic villus sampling, cervical cerclage, or amniocentesis. […] The 2021 joint guidelines from the American Society of Hematology (ASH), the International Society on Thrombosis and Hemostasis (ISTH), the National Hemophilia Foundation (NHF), and the World Federation of Hemophilia (WFH) recommend long-term prophylaxis for patients with VWD and a history of severe and frequent bleeds. […] Long-term prophylaxis for people with VWD refers to routine replacement therapy occurring at least weekly for a period of 6 months or more. […] Joint and gastrointestinal bleeding are the main indications for long-term prophylaxis in patients with type 3 VWD. […] Recommended therapies for long-term prophylaxis in type 3 VWD are pdVWF and rVWF. […] Routine prophylaxis with plasma-derived FVIII/VWF concentrate was effective and well tolerated to prevent recurrent joint bleeding, hematoma, and ear, nose, and throat bleeding. […] Plasma-derived VWF is an effective prophylactic agent; a study of children and adolescents with VWD and clinically relevant bleeding found that prophylactic treatment stopped recurrent bleeding episodes in 31 of 32 patients. […] Anaphylactic reactions are generally rare. However, infusion should be stopped if anaphylactic symptoms occur during the infusion treatment, such as urticaria, chest tightness, or pruritus.

• #1 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 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. […] Replacement therapies. These include infusions of concentrated blood-clotting factors containing von Willebrand factor and factor VIII.

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

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

  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.

• #1 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

  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. 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.

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

  https://www.vonvendipro.com/prophylaxis

  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. […] 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 […] 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 VONVENDI® Treatment for Adults With von Willebrand Disease (VWD)

  https://www.vonvendi.com/

  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.1 VONVENDI is the first and only recombinant treatment for VWD, meaning it’s made without using human blood.2,3 Recombinant products have virtually no risk of being exposed to disease that can be spread through contamination by infected human blood.2 […] In certain adult patients with severe Type 3 VWD, VONVENDI is approved to reduce the frequency of bleeding episodes.1 […] VONVENDI is used in adults (age 18 years and older) diagnosed with von Willebrand disease to: 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.

• #1 VONVENDI® Prophylaxis Treatment

  https://www.vonvendi.com/prophylaxis

  With VONVENDI prophylaxis, you may be able to protect against frequent bleeds before they even occur. […] In a clinical trial, routine prophylaxis with VONVENDI helped reduce the number of bleeds for adults with severe Type 3 von Willebrand Disease (VWD) who were previously treated on-demand. […] Prophylaxis treatment is taken routinely to reduce the number of bleeds rather than waiting for a bleed to happen and treating it on demand. VONVENDI was tested in a clinical trial for prophylaxis treatment. […] VONVENDI can be used on a twice-weekly routine by adults with severe Type 3 VWD receiving on-demand therapy to reduce the frequency of bleeding episodes. […] VONVENDI is used in adults (age 18 years and older) diagnosed with von Willebrand disease to reduce the number of bleeding episodes when used regularly (prophylaxis) in adults with severe Type 3 von Willebrand disease receiving on-demand therapy.

• #1 Take control of von willebrand disease (VWD)

  https://wilate.com/m-en/prophylaxis/

  wilate is a VWF/FVIII concentrate that is proven to be effective in people with all types of von Willebrand disease for the management of bleeding during surgery, as prophylaxis for the prevention of bleeding, and for on-demand treatment of bleeds1-3 […] Over 15 years of clinical and real-life experience have proven the efficacy and tolerability of wilate in people with all types of von Willebrand disease for surgery, on-demand treatment and prophylaxis.1-3 […] In the European Union, wilate is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease, when desmopressin treatment alone is ineffective or contra-indicated 6

• #1 Wilate Approved for Routine Prophylaxis in Von Willebrand Disease – MPR

  https://www.empr.com/home/news/wilate-approved-for-routine-prophylaxis-in-von-willebrand-disease/

  Findings showed that routine prophylaxis with wilate resulted in an 84% reduction in mean total ABRs compared with on-demand treatment. […] The Food and Drug Administration (FDA) has expanded the approval of wilate (von Willebrand Factor/Coagulation Factor VIII Complex [human]) to include routine prophylaxis to reduce the frequency of bleeding episodes in adults and children 6 years of age and older with von Willebrand disease (VWD). […] Wilate prophylaxis for adults and children aged 6 and older with VWD promises to be life-changing for many patients, said Octapharma USA President Flemming Nielsen. Patients have been forced to live with far too many bleeding episodes while receiving on-demand treatment. Octapharma is pleased to offer patients a new therapy option that can greatly improve their quality of life.

• #1 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 expanded indication is the product of WIL-31, the largest ever prospective prophylaxis study in VWD.” […] „The FDA inspections and data review went smoothly, and we received a broad indication for prophylaxis treatment in VWD that puts us ahead of our competitors, which is very exciting.” […] „Long-term prophylaxis with VWF concentrate, as compared with on-demand treatment for bleeding, is recommended for patients with severe VWD.”

• #1 Voncento and Von Willebrand Disease (VWD)

  https://www.voncento.co.uk/hcp/voncento

  Prophylaxis and treatment of haemorrhage or surgical bleeding in adults and children with VWD, when desmopressin (DDAVP) treatment alone is ineffective or contraindicated. […] Voncento is approved for the treatment of VWD, secondary to DDAVP, across all age groups and provides reliable bleed control. […] The total number of NSB events reported from the prophylactic arm decreased from 304 to 10 bleeds (n=8), with the haemostatic efficacy rated as „excellent” for all 10 events. […] The haemostatic efficacy for all non-surgical bleeds was considered excellent or good for paediatrics (12 years of age) receiving on-demand (n=13) and prophylaxis (n=4) treatment, in both the on-demand and prophylaxis arms. […] Proven efficacy for surgical bleeds, non-surgical bleeds and prophylaxis in children.

• #1

  https://www.pagepress.org/journals/index.php/hmr/article/view/270

  Patients with severe forms of von Willebrand’s disease (VWD) may have frequent episodes of mucocutaneous bleeding and also of hemarthrosis or hematomas. […] Aim of this study was to evaluate the efficacy and safety of fixed regimens of prophylaxis with factor VIII/VWF concentrates in our cohort of VWD patients with recurrent joint and gastrointestinal (GI) bleeds. […] Prophylaxis was started because of GI bleeds in 7 patients with VWD type 3 (n=1), 2A (n=4), 2M (n=1) and 1 (n=1) and for joint bleeding only in VWD type 3 (n=4). […] Prophylaxis could stop bleeding in 8 patients and largely reduced hospitalization for blood transfusions in the remaining 3. […] Secondary long-term prophylaxis by high-and intermediate purity FVIII/VWF concentrates is effective and safe in severe forms of VWD. […] Cost-effectiveness of these prophylaxis regimens versus on demand therapy should be investigated in large prospective studies.

• #1

  https://link.springer.com/article/10.1007/s00277-007-0343-1

  The importance of prophylaxis in preventing arthropathy was recently outlined by Traivaree et al. who shifted four type 3 VWD children with severe musculoskeletal bleeds from on-demand treatment to a regular long-term prophylaxis (55 UI/kg weekly or twice weekly). […] While the literature data document that short-term prophylaxis in von Willebrand disease is safe and effective, preliminary data on long-term prophylaxis are encouraging, but large prospective studies must be performed before detailed recommendations can be made.

• #1 Long-term Prophylaxis in von Willebrand Disease – How Should It Be Given and for Whom? – touchONCOLOGY

  https://touchoncology.com/haematology/journal-articles/long-term-prophylaxis-in-von-willebrand-disease-how-should-it-be-given-and-for-whom/

  Von Willebrand disease (VWD) is a common hereditary bleeding disorder that may affect as many as 1% of the general population. […] The rationale for initiating long-term prophylaxis in VWD is that it has been successfully used in severe haemophilia, showing that it is feasible to implement early in life in a home setting and that prevention of bleeding and its consequences is possible. […] The investigators concluded that long-term prophylactic treatment in VWD is warranted in the majority of cases with type 3 disease and sometimes, depending on the clinical phenotype, for those with other types. […] The primary objectives of the VIP study are to: study the effect of prophylaxis on bleeding frequency; and establish optimal treatment regimens for joint bleeding, GI bleeding, epistaxis and menorrhagia.

• #1 Long-term Prophylaxis in von Willebrand Disease – How Should It Be Given and for Whom? – touchONCOLOGY

  https://touchoncology.com/haematology/journal-articles/long-term-prophylaxis-in-von-willebrand-disease-how-should-it-be-given-and-for-whom/

  The prospective component of the VIP study is a non-randomised multicentre study to investigate the role of prophylaxis for joint bleeding, GI bleeding, epistaxis and menorrhagia. […] The retrospective studies are being conducted in parallel with the prospective study. The retrospective studies are multicentre studies with the following objectives: to examine the effect of prophylaxis on bleeding frequency in patients currently on a prophylactic regimen; and to characterise the natural history of GI bleeding in VWD. […] The percentage reduction in bleeding rate and the length of time bleed-free during follow-up will be calculated.

• #1 von Willebrand Disease: What It Is, Types, Symptoms & Treatment

  https://my.clevelandclinic.org/health/diseases/17709-von-willebrand-disease

  Most people inherit von Willebrand disease. If your biological parents have this condition, you may inherit it from one or both of them. […] Healthcare providers can treat von Willebrand disease but they cant cure it. Most people have Type 1 or Type 2 von Willebrand disease and may only need treatment if theyre injured or need surgery. People with Type 3 von Willebrand disease may need ongoing medical treatment to manage bleeding. […] Most people with von Willebrand disease have mild or moderate symptoms. For them, living with von Willebrand disease may mean they should: Avoid activities where they may get hurt, like contact sports such as football, rugby or hockey. Tell all healthcare providers, including their dentists, that they have the disease. That way, their healthcare providers can plan how to manage bleeding after surgery or dental surgery. Avoid aspirin and drugs that contain aspirin. Avoid nonsteroidal anti-inflammatory (NSAIDs) like ibuprofen, unless a healthcare provider who knows they have von Willebrand disease tells them to take an NSAID. Avoid nutritional supplement pills that contain vitamin E, fish oil or turmeric. Consider medical alert identification. Wearing a medical alert bracelet or carrying identification may help people get appropriate medical care in an emergency.

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

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

  Oral contraceptives. In addition to preventing pregnancy, these drugs can help control heavy bleeding during menstrual periods. […] 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. […] If your condition is mild, your doctor might recommend treatment only when you’re having surgery or dental work or when you’ve had a trauma, such as a car accident. […] These self-care tips can help you manage your condition: […] To help prevent bleeding episodes, talk with your doctor before taking blood-thinning medications such as aspirin, ibuprofen (Advil, Motrin IB, others) or naproxen sodium (Aleve). […] Let your doctors or dentist know that you have von Willebrand disease before you have surgery, start a new medication or give birth.

• #1 Pediatric Von Willebrand Disease Treatment & Management: Medical Care, Consultations, Activity

  https://emedicine.medscape.com/article/959825-treatment

  Avoid medications with known antiplatelet effects. Although aspirin is rarely taken by children, over-the-counter compounds containing acetylsalicylic acid often are used by adolescents. Ibuprofen and other nonsteroidal anti-inflammatory drugs (NSAIDs) are reversible cyclooxygenase inhibitors and may cause intestinal bleeding. The risks of these and other medicines with antiplatelet effects should be considered in light of the severity of the von Willebrand disease. Provide patients with von Willebrand disease a list of prescription and nonprescription medications to avoid. This list should include the following. […] Over-the-counter medications to avoid include the following: Aspirin, Ibuprofen, Naproxen, Antihistamines, Ethanol. […] Antiplatelet agents to avoid include the following: Dipyridamole, Ticlopidine, Prescription nonsteroidal anti-inflammatory compounds.

• #1 Pediatric Von Willebrand Disease Treatment & Management: Medical Care, Consultations, Activity

  https://emedicine.medscape.com/article/959825-treatment

  Antimicrobials to avoid include the following: High-dose penicillins, Cephalosporins, Nitrofurantoin, Hydroxychloroquine. […] Cardiovascular medications to avoid include the following: Propranolol, Furosemide, Calcium channel blockers, Quinidine. […] Other agents to avoid include the following: Caffeine, Tricyclic antidepressants, Phenothiazines, Valproate, Heparin.

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

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

  If you plan to have children and have a family history of von Willebrand disease, consider genetic counseling. If you carry the gene for von Willebrand disease, you can pass it on to your offspring, even if you don’t have symptoms.

• #1 Von Willebrand Disease | Cornell University College of Veterinary Medicine

  https://www.vet.cornell.edu/departments-centers-and-institutes/riney-canine-health-center/canine-health-information/von-willebrand-disease

  Because vWD is inherited, dogs suspected of having (or carrying) vWD should not be used for breeding without genetic testing and careful consideration of mate selection. DNA screening is available for several dog breeds. […] If genetic testing determines that a dog at-risk for vWD (e.g. carrying two copies of the variant), they may be subsequently tested for the amount of vWF present in their bloodstream to better characterize the severity at that point in time. If a dog is a carrier for vWD (with only one copy of the variant present), your veterinarian may also recommend testing vWF levels, especially if there are concerns that another clotting disorder may be present.

• #1 Prophylaxis in von Willebrand Disease: Is It Time for a New Perspective?

  https://www.medscape.org/viewarticle/916786

  The goal of this activity is to discuss the management of patients with von Willebrand Disease (vWD) and examine the emerging role of prophylaxis in the management of vWD. […] Current recommendations for the management/prevention of bleeding episodes in patients with vWD […] Emerging prophylaxis strategies for patients with vWD.

• #1 Prophylaxis in von Willebrand Disease: Is It Time for a New Perspective?

  https://www.medscape.org/viewarticle/916786_sidebar2

  This activity is intended for hematologists, pediatricians, primary care physicians, nurse practitioners, nurses, and other clinicians involved in the diagnosis and management of von Willebrand disease (vWD). […] The goal of this activity is to discuss the management of patients with von Willebrand Disease (vWD) and examine the emerging role of prophylaxis in the management of vWD. […] Current recommendations for the management/prevention of bleeding episodes in patients with vWD […] Emerging prophylaxis strategies for patients with vWD. […] Prophylaxis is something we know more about in hemophilia, but less so in vWD. […] There are a number of retrospective reviews of vWD prophylaxis out there that demonstrate you can reduce the bleeding if you start vW patients with severe bleeding, such as recurrent nosebleeds or recurrent current joint bleeds, on prophylaxis.

• #1 Breaking new ground in von Willebrand disease treatment

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

  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. […] A clinical trial is underway for both von Willebrand and hemophilia A. […] 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. […] 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.

• #1 Paper: The Potential Role of Emicizuamb Prophylaxis in Severe Von Willebrand Disease

  https://ash.confex.com/ash/2020/webprogram/Paper138777.html

  Severe Von Willebrand’s disease (VWD) may be associated with chronic joint damage and may require prophylactic therapy. […] This is the first study assessing the potential role of emicizumab as an alternative prophylactic treatment in a cohort of patients with severe VWD. […] Prophylactic treatment was administered in the majority of type 3 VWD patients, whereas type 2 VWD patients largely required only intermittent on demand therapy applied for bleeding episodes or any surgical interventions. […] Our data suggests that some severe VWD patients could be safely and efficiently treated with emicizumab. The successful prophylaxis of our patient and our ex vivo laboratory findings should set the ground for further collaborative multicenter studies to examine the efficacy and safety of emicizumab prophylaxis in type 3 VWD patients. […] OffLabel Disclosure: The use of Emicizumab in sever Von Willebrand disease.

• #1 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). […] A notable advantage of VGA039 is its subcutaneous administration, which contrasts with the intravenous infusions required by current von Willebrand disease prophylaxis therapies. This method has the potential to reduce the treatment burden for patients. […] 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. […] While still in the early stages of development, the recent Fast Track designation indicates that Star Therapeutics might have a new, more convenient solution for von Willebrand disease.

• #1 Wilate® is now approved in Canada for Prophylaxis in Von Willebrand Disease

  https://www.octapharma.ca/en/news/press-release/2024/wilate-is-now-approved-in-canada-for-prophylaxis-in-von-willebrand-disease

  Prophylaxis treatment has been shown to significantly improve patient outcomes, reducing the frequency and severity of bleeding episodes and enhancing overall quality of life. Patients can expect fewer interruptions in their daily activities, less time spent in hospitals, and a greater sense of normalcy in their lives. This proactive approach to managing VWD represents a monumental shift in patient care, promising a brighter future for those living with this condition.

• #1 Prophylaxis in Von Willebrand Disease: Insights from Dr. Chai Phua

  https://www.linkedin.com/pulse/prophylaxis-von-willebrand-disease-insights-from-dr-drdzc

  The goal of prophylaxis is to minimize the disease burden by preventing bleeding events associated with VWD. This allows patients to participate fully in their daily lives without the disease impacting their activities. Prophylaxis personalizes medicine by tailoring treatment to reduce bleeding risks, thus enhancing patients quality of life. […] This approval marks a significant advancement in hematology, emphasizing the broader application of prophylaxis in Von Willebrand Disease. By preventing bleeding, we can fundamentally change how we treat patients, allowing them to live their best lives without VWD impacting their daily activities. The significance of prophylaxis in VWD management points to a paradigm shiftmoving from treating bleeding events to preventing them. This approach helps improve patients quality of life, enabling them to engage in daily activities with less anxiety about physical activities, fewer bleeding events, less time off work, and fewer hospitalizations. All of this contributes to the enhanced quality of life that has evolved in VWD management.

• #1 Effective Treatments for Von Willebrand Disease: Guidelines and Options | Hemophilia Outreach Center

  https://hemophiliaoutreach.org/effective-treatments-for-von-willebrand-disease-guidelines-and-options/

  Individuals with VWD should avoid medications that can increase bleeding risk, such as nonsteroidal anti-inflammatory drugs (NSAIDs) like ibuprofen and aspirin. […] Emergency preparedness is crucial for individuals with VWD. Having an emergency treatment plan, including a list of medications, a medical alert bracelet, and contact information for healthcare providers, can be lifesaving in situations where immediate medical attention is required. […] Treatment plans for VWD are often customized to the individual, taking into consideration factors like age, lifestyle, and the specific type of VWD. […] Regular check-ups are essential for individuals with VWD, as they allow healthcare providers to monitor treatment efficacy and make necessary adjustments.

• #1 VONVENDI® Dosing for Prophylaxis, On-Demand, and Surgery

  https://www.vonvendipro.com/dosing

  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. […] Consider prophylaxis for your appropriate adult patients receiving on-demand therapy. […] Dosing of VONVENDI, with or without rFVIII, should be based on patient need as determined by monitoring levels and clinical judgment. […] VONVENDI is the first and only recombinant VWF for prophylaxis.

• #1 wilate® Official Website – VWD & Hemophilia A Treatment Optionwilate-logo

  https://wilateusa.com/

  Indications wilate is a von Willebrand Factor/Coagulation Factor VIII Complex (Human) indicated in children and adults with von Willebrand disease for on-demand treatment and control of bleeding episodes; for perioperative management of bleeding; and for routine prophylaxis to reduce the frequency of bleeding episodes in children 6 years of age and older and adults with VWD. […] Monitor plasma levels of VWF:RCo and FVIII activities in patients receiving wilate to avoid sustained excessive VWF and FVIII activity levels, which may increase the risk of thromboembolism, particularly in patients with known clinical or laboratory risk factors. […] Monitor for development of VWF and FVIII inhibitors. Perform assays to determine whether VWF and/or FVIII inhibitor(s) is present if bleeding is not controlled with the expected dose of wilate.

• #2 Prophylactic management of patients with von Willebrand disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8721401/

  With this background, this narrative review focuses on the short-term and long-term prophylactic management of VWD patients. […] The choice of the best treatment regimen for short-term prophylaxis lies among desmopressin (DDAVP), VWF-FVIII plasma-derived products, and products containing only VWF (plasma-derived or produced by recombinant DNA technology). […] Another potential application of short-term prophylaxis in VWD is for the management of pregnant women at the time of labor with the goal of avoiding postpartum hemorrhage. […] The definition of long-term prophylaxis encompasses replacement therapy administered once weekly and for a period of at least 6 months. VWD warrants long-term prophylaxis mainly in cases with a severe bleeding phenotype (i.e. hemarthroses with target joints and arthropathy). In such individuals, who generally have type 3 and more seldom severe type 1 VWD, long-term prophylaxis may be the optimal therapeutic approach instead of on-demand treatment when the occasion is a bleed. […] Collectively, the results of these studies document the feasibility and efficacy of secondary long-term prophylaxis in VWD.

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

  https://www.octapharma.com/news/innovation-and-science/2024/wil-31-expanding-treatment-options-for-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.” […] „The primary endpoint of the study, a greater than 50% reduction in mean total annualised bleeding rate (ABR) during von Willebrand factor prophylaxis compared with prior on-demand treatment, was met.” […] „In fact, the primary endpoint of the study was met with an 84% reduction in the mean total annual bleeding rate (ABR) compared with on-demand treatment during the previous study.” […] „Putting patients at the centre of its activities has always been a top priority for Octapharma, where clinical trials and studies have long been directed towards reducing high levels of unmet patient need.”

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