Kucher, A., Skinner, M. W., Clearfield, E., Brennan, F., Rotellini, D., Colle, Y., & Wilton, P. A. (2025). Development of a WGPPM-Focused PROBE Survey: Addressing underrepresentation in patient-reported outcomes and quality of life research [Poster presentation]. National Bleeding Disorders Foundation (NBDF) Annual Conference, Atlanta, GA, United States.
Objective
Women, Girls, and People with the Potential to Menstruate (WGPPM), including those with von Willebrand disease (VWD), platelet function disorders, rare coagulation factor deficiencies, and other rare bleeding disorders, remain significantly underrepresented in research related to patient-reported outcomes (PROs) and health-related quality of life (HRQoL). Existing tools such as the Patient Reported Outcomes, Burdens, and Experiences (PROBE) survey—originally developed in 2012—have primarily focused on individuals with hemophilia A or B and carriers, with data also collected from individuals without a bleeding disorder (NoBD) serving as a control group. However, the unique experiences, burdens, and health impacts specific to WGPPM have not been fully captured in existing survey instruments.
Methods
To address this gap, a targeted literature review using PubMed was conducted to identify key concepts influencing the quality of life in WGPPM. In parallel, the study team contributed historical examples of related surveys for review. These sources were analyzed to identify novel domains and items, which were then organized into a conceptual framework. This framework was compared against the existing PROBE questionnaire to assess gaps in content coverage. Based on this analysis, the PROBE instrument was revised to include concepts relevant to WGPPM. The development process was undertaken in collaboration with key stakeholders, including the World Federation of Hemophilia, Coalition of the Americas, European Haemophilia Consortium, and the National Bleeding Disorders Foundation, all of whom contributed to a dedicated working group guiding this initiative.
Summary
The updated framework expands the PROBE instrument with an additional 13 questions which include several WGPPM-specific concepts, such as access to healthcare (including specialty care), emergency department utilization, and bleeding episodes unique to this population, including menstruation and gynecological complications. Additional domains address reproductive decision-making, including considerations around family planning, as well as the social and emotional impact of living with a bleeding disorder—particularly as it pertains to leisure activities and interpersonal relationships. Furthermore, the revised tool allows for improved representation of individuals with rare and often overlooked bleeding disorders.
Conclusion
The WGPPM-specific PROBE exploratory pilot was launched in early 2025 through an online platform. It is anticipated that this pilot will generate comprehensive and representative data to better understand the lived experiences of WGPPM. These findings will inform future efforts in research, advocacy, clinical care, and health policy, while also serving as the basis for subsequent updates to the PROBE questionnaire to ensure its continued inclusivity and relevance.
Abstract: HERE
