A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers

The main aim of this study is to describe the experience and unmet needs of persons living with VWD and their caregivers in Canada.The survey is planned to be done in two phases: The first phase will be directed at adult participants; the second phase will focus on children and teenagers. At the end of the first phase the Sponsor will decide if the second phase will be started.Participants and their caregivers will be asked to answer a set of questions either using an online questionnaire or through interviews. The participant/caregiver's perception, experience, satisfaction, and unmet needs, and need for new treatments or new indications will be determined based on their responses to the questions.

Inclusion criteria:Phase 1:Adult participants (age ≥18 years) with severe VWD (self-BAT ≥10)Participants who have received von Willebrand factor (VWF) treatment, either for on-demand treatment, regular prophylaxis, or situational prophylaxis (e.g., surgery) within the last 5 yearsFor caregivers: Current caregiver of participants with severe VWDFor caregivers and participants:Fluent in English or FrenchConsent to participate in an individual phone interview and to fill self-administered questionnairesAdditional inclusion criteria for virtual focus groups:Access to technology (Internet and email)Consent to participate in a virtual focus group with an audio recording of the session.Phase 2:Same as above for participants pediatric participants (age <18 years) with severe VWD (self-PBQ score of ≥3 for at least one symptom).Exclusion criteria:Phase 1:Participants or caregivers of participants who do not have severe symptoms of VWDPediatric participants (age <18 years)Participants who have not received any treatment (on-demand, regular or situational prophylaxis) within the last five yearsParticipants who are successfully treated with desmopressin or anti-fibrinolytic medicationsParticipants with inherited or acquired hemostatic or bleeding disorders other than congenital VWD (self-reported)Participants and caregivers of participants who show cognitive impairment (as assessed by the research nurse at the time of screening)Participants and caregivers who are qualified as health care practitioners currently working in a health-care capacity (e.g., physician, nurse, or healthcare aid)Participants or caregivers who do not reside in CanadaCurrent or past participation within the last 12 months in a clinical trialPhase 2: Same as above excluding participants of ≥18 years of age