The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD). Data from this study will be used to establish baseline bleeding and treatment rates in a population of participants with VWD and act as comparator data for future clinical study outcomes.(e.g. Velora Pioneer)
Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations.
Has an understanding, ability, and willingness to comply with Study procedures and restrictions.
Is 16 years and < 70 years at the time of screening.
Weight 50 to 120 kg (±10%) at Screening and body mass index (BMI) <38.5 kg/m*2.
Has a history of clinically significant hypersensitivity associated with monoclonal antibody therapies.
Has a personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial vein thrombosis events.
Has a high-risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL/prothrombin gene mutation, antithrombin <50%, congenital protein C and protein S deficiency with levels <50%.
Requires ongoing hemostatic (bleed-prophylaxis) treatment to prevent bleeding
Participants ≥16 years of age with VWD.
Cohort
Prospective
Clinical outcomes of patients with VWD, Type 1
Other interventionAccumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 1
Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3
Other interventionAccumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeding events in participants with VWD, Type 2A, Type 2M, Type 2N and Type 3.
Annualized bleeding event rate
Annualized treated bleeding rate
Annualized heavy menstrual bleed rate
Number of overnight admissions
Hospitalization monitoring of bleeding events.
Prophylactic and on demand treatment
Details of treatment used for bleeding events
Iron status
Blood samples will be collected for the determination of ferritin and hemoglobin levels.
Patient-Reported Outcomes Measurement Information System (PROMIS)-29
An assessment which evaluates four items from each of the seven PROMIS domains. Each question is rated on a scale of 1 to 5. In addition, the PROMIS-29 includes one pain intensity question which is rated on a scale of 1 to 10. Higher scores on the PROMIS-29 indicate worse symptoms.
Menstrual Bleeding Questionnaire (MBQ)
Measure the effect of heavy menstrual bleeding on a self-assessment of menstrual blood loss, limitations in social and leisure activities, physical activities, and work activities.
