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

Completed

Brief 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.

Detailed Description: 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.

Recruitment & Eligibility

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
Phase 1: Adult Participants	No Intervention	Adult participants with severe VWD (self-bleeding assessment tool \[BAT\] score ≥10) and their caregivers will be enrolled as per protocol specifications. Data will be collected from participants via questionnaire, semi-structured interviews, and focus groups using a virtual platform.
Phase 2: Pediatric Participants	No Intervention	Pediatric participants with severe VWD (self-pediatric bleeding questionnaire \[PBQ\] score of ≥3 for at least one symptom and caregivers will be enrolled as per protocol specifications. Data will be collected from participants via questionnaire, semi-structured interviews, and focus groups using a virtual platform. The decision to proceed with Phase 2 will be determined following completion of Phase 1.

Primary Outcome Measures

Name	Time	Method
Number of Participants Categorized by Impact on Daily Life	Up to approximately 9 months	The age-adapted impact on daily life will be categorized by questions related to quality of life, physical activity, professional life, school, financial impact, mental health, relationships, avoidance of social and physical activities, and impact on daily activities.
Number of Participants Categorized Based on Bleeding Characteristics	Up to approximately 9 months	Bleeding characteristics will include categories of bleed frequency, bleed type/location and bleed severity.
Number of Participants Categorized Based on Disease Management	Up to approximately 9 months	Disease management will be categorized by need for subsequent therapies, need for additional investigations, time needed for disease management, impact on future planning and treatment access for aging participants.

Secondary Outcome Measures

Name	Time	Method
Time (Delay) to Treatment Initiation	Up to approximately 9 months
Number of Participants Who Missed Days at Work/School	Up to approximately 9 months	The number of participants who missed days at work/school will include categories for patient and caregiver.
Duration of Therapy Schedule	Up to approximately 9 months
Participant's Experience Assessed as Number of Participants Categorized Based on Symptom Severity and Comorbidities Over Time	Up to approximately 9 months
Number of Participants With Change in Treatment Frequency	Up to approximately 9 months
Time to Bleed Control	Up to approximately 9 months
Treatment Experience Based on Number of Participants Satisfied With the Treatment	Up to approximately 9 months
Number of Participants With Bleed Control	Up to approximately 9 months
Duration of Inpatient and Outpatient Hospital Visits	Up to approximately 9 months