Kucher A, Skinner M. W., Clearfield E., Brennan F., Rotellini D., Colle Y., Wilton P. A. Development of Women, Girls, and People Who Have or Had the Potential to Menstruate (WGPPM) questionnaire for PROBE study. (2025), Book of Abstracts. Haemophilia, 31: 21-109. https://doi.org/10.1111/hae.70032
Objective
The Patient Reported Outcomes, Burdens, and Experiences (PROBE) survey was first developed in 2012. PROBE currently collects data from people with hemophilia A or B, including carriers, and people without a bleeding disorder (NoBD) who serve as a control group. To date, there has been limited global comparative patient reported data on health outcomes and quality of life (HRQoL) about WGPPM.
Methods
A targeted PubMed literature review was conducted to identify concepts impacting WGPPM quality-of-life. The study team also provided additional examples of past surveys for review. Novel topics and items were mapped to a conceptual framework. The resulting framework was compared to the PROBE questionnaire and subsequently PROBE was updated to include missing concepts. It is anticipated that this exploratory questionnaire will lead to an expanded PROBE to more comprehensively include WGPPM. The questionnaire was developed in consultation with the World Federation of Hemophilia, Coalition of the Americas, European Haemophilia Consortium and National Bleeding Disorders Foundation who constituted the project working group. The new WGPPM questionnaire will be initiated as an on-line survey.
Results
The group identified von Willebrand Disease (VWD), platelet disorders, rare factor deficiencies, and other rare bleeding disorders for inclusion. New concepts include access to healthcare (including specialties); visits to emergency room; bleeding episodes unique to WGPPM (e.g., menstrual bleeding; gynecological problems), decision-making around having children, and impact on social and leisure life.
Conclusions
To advance management and treatment for this underserved population it is crucial to collect and report comprehensive data on WGPPM health outcomes and quality of life. The WGPPM PROBE exploratory pilot was launched in early 2025. Through this pilot we anticipate more accurate and comprehensive data will be available to support future advocacy, research, management and treatment. Insights from the pilot will be applied to future PROBE updates.
Poster: HERE
