From the Guidelines
Von Willebrand disease treatment should prioritize desmopressin (DDAVP) as the first-line therapy to increase von Willebrand factor (VWF) levels and control bleeding, as supported by the most recent and highest quality study 1.
Treatment Overview
The primary goal of treating Von Willebrand disease is to increase VWF levels to control bleeding. Desmopressin (DDAVP) is the primary treatment, which stimulates the release of stored VWF from endothelial cells.
- DDAVP can be administered intravenously at 0.3 mcg/kg, intranasally as Stimate at 150-300 mcg per dose, or subcutaneously.
- For minor bleeding, a single dose may be sufficient, while more severe bleeding may require doses every 12-24 hours for 2-3 days.
- Patients should be monitored for hyponatremia, especially with multiple doses.
Alternative Therapies
For those who don't respond to DDAVP or have type 3 disease, VWF replacement therapy with products like Humate-P, Wilate, or Alphanate is recommended.
- The dosing is typically 40-60 IU/kg initially, followed by 20-40 IU/kg every 8-24 hours as needed.
- For menorrhagia, hormonal therapies like oral contraceptives can be effective.
- Antifibrinolytic agents such as tranexamic acid (1-1.5 g orally 3-4 times daily) or aminocaproic acid can be used as adjunctive therapy.
- Patients should avoid aspirin and NSAIDs which can worsen bleeding tendencies.
Individualized Treatment
Treatment should be individualized based on the disease type, severity, and specific bleeding episode, as emphasized by studies 1.
- The use of desmopressin has been evaluated in various settings, including peri-operative and trauma cases, with mixed results.
- However, the most recent study 1 provides the strongest evidence for the use of desmopressin in Von Willebrand disease treatment.
From the FDA Drug Label
Desmopressin acetate should not be used to treat patients with Type IIB von Willebrand’s disease since platelet aggregation may be induced. Desmopressin acetate can be used to treat Von Willebrand disease, but not Type IIB.
- Contraindication: Type IIB von Willebrand’s disease 2
From the Research
Treatment Options for Von Willebrand Disease
- Desmopressin is a common treatment for type 1 von Willebrand disease, as it can induce the release of von Willebrand factor (VWF) from cellular compartments 3, 4.
- Plasma concentrates containing VWF and factor VIII (FVIII) are used in cases where desmopressin is not effective or is contraindicated, such as in type 3 von Willebrand disease or in some cases of type 2 disease 3, 5, 6, 4.
- Antifibrinolytics may be used as adjunct therapies to prevent bleeding 5.
Desmopressin Treatment
- Desmopressin can increase VWF activity by 452% with an EC50 of 0.174 ng/ml 7.
- A dose of 0.3 mcg/kg desmopressin intravenously can achieve a VWF activity >0.5 IU/ml in >90% of patients with a baseline VWF activity ≥0.20 IU/mL 7.
- Desmopressin is not effective in type 3 von Willebrand disease and in severe forms of type 1 and 2 disease 3, 6, 4.
Plasma Concentrates
- Plasma concentrates containing VWF and FVIII can be used to replace these factors in patients with von Willebrand disease 5, 6, 4.
- Different formulations of VWF and FVIII concentrates are available, and the choice of product may depend on the patient's specific needs and the availability of products in different regions 5.