Does Von Willebrand Disease (VWD) require treatment?

From the Guidelines

Von Willebrand Disease (VWD) does require treatment, but the approach depends on the type and severity of the condition, with many mild cases requiring no treatment at all. For those who do need treatment, options include desmopressin (DDAVP), which can be administered as a nasal spray (Stimate) or intravenously at doses of 0.3 mcg/kg to stimulate the release of stored von Willebrand factor, as supported by studies such as 1. Von Willebrand factor concentrates (such as Humate-P, Alphanate, or Wilate) may be necessary for more severe cases or when DDAVP is ineffective, typically administered intravenously based on weight and severity.

Treatment Options

• Desmopressin (DDAVP) can be used to stimulate the release of stored von Willebrand factor, with a recommended dose of 0.3 mcg/kg, as seen in studies like 1.
• Von Willebrand factor concentrates may be used for more severe cases or when DDAVP is ineffective.
• Antifibrinolytic medications like tranexamic acid (1000-1500 mg three times daily) or aminocaproic acid can help stabilize clots.
• Hormonal treatments like birth control pills may help manage symptoms during menstruation.

Considerations for Treatment

The goal of treatment is to correct the dual defect in VWD: low levels of von Willebrand factor (which helps platelets stick to damaged blood vessels) and potentially reduced Factor VIII activity (needed for the clotting cascade), thus preventing or controlling bleeding episodes, as outlined in guidelines such as those referenced in 1. Treatment is typically given before surgical procedures or dental work and during active bleeding episodes. It's also important to consider the patient's stress level, recent exercise, and other conditions that may affect VWF and FVIII levels, as discussed in 1.

Diagnosis and Classification

VWD can be classified into different types, including Type 1, Type 2 (with subtypes 2A, 2B, 2M, 2N), and Type 3, each with distinct characteristics and treatment approaches, as described in 1. Accurate diagnosis and classification are crucial for determining the best course of treatment.

Laboratory Evaluation

Laboratory testing, including VWF:Ag, VWF:RCo, and FVIII assays, is essential for diagnosing and managing VWD, with calibrators referenced to the World Health Organization (WHO) plasma standard, as mentioned in 1. The quality of laboratory testing can vary, and test results should be interpreted in the context of the patient's clinical presentation and medical history.

Clinical Evaluation

A thorough clinical evaluation, including a detailed medical history and physical examination, is necessary to assess the patient's bleeding symptoms and risk factors, as outlined in 1. This information, combined with laboratory test results, will guide treatment decisions and ensure the best possible outcomes for patients with VWD.

From the FDA Drug Label

Desmopressin acetate injection 4 mcg/mL is indicated for patients with mild to moderate classic von Willebrand’s disease (Type I) with factor VIII levels greater than 5% Desmopressin acetate injection will often maintain hemostasis in patients with mild to moderate von Willebrand’s disease during surgical procedures and postoperatively when administered 30 minutes prior to the scheduled procedure Desmopressin acetate injection will usually stop bleeding in mild to moderate von Willebrand’s patients with episodes of spontaneous or trauma-induced injuries such as hemarthroses, intramuscular hematomas or mucosal bleeding

Von Willebrand Disease (VWD) may require treatment, specifically for patients with mild to moderate classic von Willebrand’s disease (Type I) with factor VIII levels greater than 5%. Treatment with desmopressin acetate injection can help maintain hemostasis during surgical procedures and postoperatively, and can also stop bleeding in patients with episodes of spontaneous or trauma-induced injuries. However, severe classic von Willebrand’s disease (Type I) is not indicated for treatment with desmopressin acetate injection 2.

From the Research

Treatment of Von Willebrand Disease (VWD)

• VWD is a heterogeneous disorder, and the need for treatment varies among patients, with some having no significant bleeding symptoms throughout their lives 3.
• Patients with moderate to severe factor VIII (FVIII) and von Willebrand factor (VWF) deficiency usually require treatment to stop or prevent bleeding 3.
• Treatment options include desmopressin administration to increase autologous FVIII/VWF and infusion of plasma-derived concentrates to provide normal allogeneic VWF 3, 4.

Treatment Approaches

• Desmopressin is often used as a first-line treatment, but its effectiveness depends on the patient's subtype and response to the medication 3, 5.
• Plasma-derived VWF-containing FVIII concentrates are used in patients unresponsive to desmopressin, with dosing based on FVIII content or VWF functional activity 4, 5.
• Recombinant VWF concentrate has recently become available, offering a new treatment option for patients with severe VWD 6.

Prophylaxis in VWD

• Prophylaxis is not as commonly used in VWD as it is in severe hemophilia, but recent studies suggest its value in preventing frequent bleeding in patients with severe disease 6.
• Prophylactic treatment with VWF concentrate may be considered the standard of care for patients with severe VWD, particularly those with type 3 VWD 6.

Surgical Settings

• Guidelines for managing patients with VWD in surgical settings recommend using VWF/FVIII concentrates based on the analysis of their content and pharmacokinetic studies 5.
• Treatment with highly purified, doubly inactivated FVIII/VWF concentrates, such as Fanhdi, has shown excellent clinical efficacy in bleeding episodes and surgery 7.