a survey to describe the experience and unmet needs of persons living with von willebrand disease (vwd) and their caregivers

Conditions: Von Willebrand Disease (VWD)

Summary: 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.

this study is a non-interventional, prospective, qualitative survey to know the unmet needs of participants living with vwd and their caregivers. the study will enroll approximately 49 patients, taking into scope both the participant's and caregiver's perspectives, and is planned to be conducted in two phases: phase 1: adult participants phase 2: pediatric participants the decision to proceed with phase 2 will be determined at the completion of phase 1. this multi-center trial will be conducted in canada. the overall time for data collection in this study is approximately 9 months.

Eligibility criteria:
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 years * for caregivers: current caregiver of participants with severe vwd * for caregivers and participants: * fluent in english or french * consent to participate in an individual phone interview and to fill self-administered questionnaires * additional 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 vwd * pediatric participants (age \<18 years) * participants who have not received any treatment (on-demand, regular or situational prophylaxis) within the last five years * participants who are successfully treated with desmopressin or anti-fibrinolytic medications * participants 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 canada * current or past participation within the last 12 months in a clinical trial * phase 2: same as above excluding participants of ≥18 years of age

Keywords: drug therapy