Clearfield E, Brennan F, Kucher A, Skouw-Rasmussen N, Ziemele B, Rotellini D, Delaney M, Curtis RG, O’Mahony B, Page D, Iorio A, Germini F, Skinner MW. Adapting the Patient Reported Outcomes Burdens and Experiences (PROBE) Study to Measure Quality of Life in People With von Willebrand Disease (VWD).Poster presented at: WFH 2026 World Congress; 2026 Apr 19–22; Kuala Lumpur, Malaysia. Poster PP-322.
Introduction
The coreVWD Initiative identified a core outcome set for clinical trials for von Willebrand disease (VWD); quality of life (QoL) was included as a core outcome. There is currently no disease-specific patient reported outcome measure (PROM) to assess QoL for people with VWD (PwVWD). A mapping exercise of the coreVWD results to the Patient Reported Outcomes Burdens and Experiences (PROBE) study identified coverage of some core outcomes, but gaps remained on key outcomes agreed to by the multi-stakeholder panel. PROBE is an established QoL survey currently available only for people with hemophilia, carriers, and healthy controls. Our objective is to adapt PROBE for use by PwVWD by creating a VWD-specific section and updating current items to reflect the experience of living with and managing VWD.
Methods
This is a protocol for a qualitative study. PROM development consists of semi-structured interviews with PwVWD, which will be recorded and transcribed. Thematic analysis will be performed to develop a conceptual framework for QoL for PwVWD. The framework will be mapped to the current PROBE instrument to identify which concepts are already included and what needs to be added in a VWD module. New/updated items will be tested in cognitive debriefing interviews, with a focus on relevance and respondent understanding to move to a content validated adapted instrument. This work is planned as a two-year project with the updated instrument available at the end of Year 1. In Year 2, the adapted questionnaire will undergo online programming, translation, and further psychometric testing such as test-retest and face validity studies.
Results
Ethical approval has been obtained. Recruitment for interviews has begun. A thematic overview of new concepts and items requiring adaptation will be presented.
Conclusion
As more clinical trials for new products and expanding indications are initiated, the adapted PROBE instrument will be an important tool to measure QoL in PwVWD and use in registries such as the World Bleeding Disorders Registry (WBDR). Data from the robust, established PROBE database can serve to benchmark on key QoL indicators, such as pain and interference in activities of daily living, and compare to PROBE scores from people with hemophilia.
Poster: HERE
