Study of Emicizumab Injection to Prevent Bleeding in Severe Von Willebrand Disease and Combined VWDHemophilia A Patients

What this Trial Is About

Von Willebrand Disease VWD is the most common inherited bleeding disorder, affecting up to 0.1% of people. It often causes mucous membrane bleeding, heavy menstrual bleeding, surgical bleeding, or other bleeding challenges. Severe cases require treatment with von Willebrand factor VWF concentrates given intravenously. This study is evaluating emicizumab, a monoclonal antibody given by subcutaneous injection, as a potential prophylactic treatment for severe VWD and patients with both VWD and hemophilia A. Participants will receive emicizumab starting with a loading dose of 3 mgkg once weekly for 4 weeks, followed by a maintenance dose of 1.5 mgkg once weekly for one year. Dose escalation to 3 mgkg weekly may occur after 24 weeks if bleeding control is insufficient. Breakthrough bleeding can be treated with usual therapies such as antifibrinolytics or VWFFVIII concentrates. The study is open-label and will collect treatment records and bleeding logs throughout. Participants will be monitored for efficacy and safety over 18 months, including patient-reported outcomes on quality of life, physical, emotional, social, and general symptoms. Data on annualized bleed rates and treatment burden compared to previous therapies will be collected. Safety assessments and adherence to emicizumab prophylaxis will be tracked as well. This includes a one-year retrospective review of bleeding history before starting treatment.

Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A