Emicizumab for Severe VON Willebrand Disease (VWD) and VWD/Hemophilia A
Bleeding and Clotting Disorders Institute Peoria, Illinois
Summary
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. Severe bleeding events require VWF concentrates administered solely through intravenous access. Emicizumab (Hemlibra) is a monoclonal bispecific antibody developed to bind activated FIX and FX and mimic FVIII cofactor functionality. Hemlibra is administered via subcutaneous injection rather than intravenous infusion. The hypothesis of this study is that Emicizumab is safe and efficacious for prophylaxis in severe VWD and concomitant VWD/hemophilia patients.
Description
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. VWD currently has few therapies generally useful in management of bleeding events including antifibrinolytics, desmopressin (DDAVP), and VWF concentrates, which may be plasma-derived (VWF with and without FVIII) or recombinant. Minor bleeding may be successfully treated with antifibrinolytics and DDAVP; however, more severe bleeding requires VWF concentrates that are adminis…
Eligibility
- Age range
- 0–90 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Signed informed consent * Age 0 and older (infants weighing ≥3 kg) * ability to comply with protocol in investigators judgement * diagnosis of: severe VWD type 3, or VWD with VWF antigen, activity or collagen binding \</= 20 U/dl or variant VWD confirmed by genetic mutation and VWF ag, activity or CB \< 50 U/dl based on historical medical records of study site. * diagnosis of VWD/hemophilia A defined as VWF:ag, activity or CB \<50 U/dl, and mild moderate or severe hemophilia A(defined by ISTH criteria) based on historical medical records of the study site. * plan to be a…
Interventions
- DrugEmicizumab
Subcutaneous injection of emicizumab for prophylaxis
Locations (12)
- The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders (CIBD)Orange, California
- Stanford University: Stanford Children's HealthRedwood City, California
- University of Miami - Miller School of MedicineCoral Gables, Florida
- St. Joseph's Children's Hospital - Center for Bleeding and Clotting DisordersTampa, Florida
- Bleeding and Clotting Disorders Institute (BCDI)Peoria, Illinois
- Innovative Hematology, Inc. (IHI)Indianapolis, Indiana