Abstracts

Brennan F, Clearfield E, Kucher A, Colle Y, Brito del Pino Mouro A, Wilton P, Skouw-Rasmussen N, Ziemele BM, Rotellini D, Delaney M, Curtis RG, O’Mahony B, Page D, Iorio A, Germini F, Skinner MW. Advancing global data collection for women with bleeding disorders: early insights from the PROBE WBD module. Haemophilia. 2026;32:4-224. Abstract PP-399. doi:10.1111/hae.70232.

Introduction

Since 2014, the PROBE (Patient Reported Outcomes, Burdens, and Experiences) Study has generated global patient-reported data to compare health outcomes and quality of life (QoL) across people with haemophilia and control populations. However, PROBE was originally designed primarily for men, leaving a substantial knowledge gap for women with bleeding disorders (WBD), including women and girls with haemophilia A or B, carriers, and those who have or had the potential to menstruate. Limited comparative Quality of Life (QoL) data, together with a longstanding pattern of underdiagnosis and misdiagnosis, highlight the need for a dedicated data-collection framework. This project aims to expand PROBE to better capture the lived experiences and health outcomes of WBD.

 Methods 

Building on earlier PROBE analyses, a structured enhancement process began in 2024. A targeted literature review and conceptual framework exercise identified domains insufficiently captured in the current PROBE instrument, including menstrual bleeding patterns, gynaecological complications, access to care, emergency room utilisation, reproductive decision-making, and impacts on social and leisure activities. Eleven new WBD-specific questions were developed and integrated following consultation with a global expert working group representing the World Federation of Hemophilia (WFH), European Haemophilia Consortium (EHC), Coalition of the Americas (CoA), National Bleeding Disorders Foundation (NBDF), and Canadian Hemophilia Society (CHS). Of the 64 pilot participants, 33% completed the WBD module. The module launched as an online exploratory pilot on International Women’s Day 2025.

 Results

Preliminary data reveals significant unmet needs among WBD. Participants reported a mean age of diagnosis of 20.45 years, with some reporting up to nine emergency department visits in the previous 12 months. The reported average menstrual period length was 10.6 days. Respondents also described moderate to significant impacts of their bleeding diagnosis and symptoms on decisions regarding childbearing and participation in social or leisure activities. Early findings from carriers, individuals with factor and platelet disorders, and those with extremely rare bleeding disorders underscore the diversity and severity of challenges experienced.

 Conclusion 

Early insights from the WBD module reinforce the necessity of integrating a dedicated WBD component into the core PROBE study. Enhanced, inclusive data collection will support more accurate understanding and inform targeted advocacy, research, management, and treatment strategies for this underserved population.

Poster: HERE

Svirca I, Kukaj E, Svirca L, Rotellini D, Harvey-Simi K, et al. Patient-reported outcomes in hemophilia in Kosovo: results from the PROBE questionnaire. Haemophilia. 2026;32:4-224. Abstract PP-317. doi:10.1111/hae.70232.

Introduction

National-level patient-reported outcomes (PROs) for people with hemophilia (PWH) in Kosovo are limited. The PROBE (Patient-Reported Outcomes, Burdens and Experiences) questionnaire captures treatment patterns, pain, function, and health status.

The aim is to characterize the Kosovo cohort with PROBE and compare key outcomes between severe and moderate/mild hemophilia contextualized against a local control group with no bleeding disorder (PwNBD).

 Methods 

We conducted a cross-sectional analysis of participants in Kosovo who completed PROBE during the National Bleeding Disorders Foundation and Kosovo Hemophilia Foundation (KHF) World Federation of Hemophilia (WFH) twinning program. A formal framework of cooperation between KHF, WFH, and the Ministry of Health of Kosovo was executed. Data were collected between April 2023 and May 2024. Outcomes included treatment regimen, acute and chronic pain, missed work or school, EQ-5D index, and PROBE total score. Participants were grouped as Severe, Moderate/Mild, or PwNBD, and results were summarized descriptively.

 Results

Thirty-three participants were included in the initial pilot data collection: Severe (n=6), Moderate/Mild (n=13), and No BD/Carriers (n=12). Acute pain was reported by 66.7% of Severe versus 46.2% of Moderate/Mild participants; chronic pain by 50.0% versus 53.9%. Mean days missed from work or school in the previous six months were higher in the PWH group than in PwNBD. The EQ-5D index (mean) was 0.558 in Severe, 0.859 in Moderate/Mild, and 0.950 in PwNBD. PROBE total scores (mean) were 0.794, 0.843, and 0.906, respectively, indicating worse reported outcomes in hemophilia groups versus controls. Additional patient characteristics are described in Table 1.

 Conclusion 

Using the PROBE instrument to measure quality of life, individuals with severe hemophilia in Kosovo reported lower health status (EQ-5D) and higher burden (PROBE score) with greater functional impact (missed work/school) than those with moderate/mild disease, and both groups fared worse than the PwNBD control group. Only one-third of Severe participants were on regular prophylaxis, with half relying on intermittent regimens and some reporting no treatment availability. Findings highlight persistent unmet needs in access to regular prophylaxis, pain management, and joint health. Ongoing data collection will support longitudinal evaluation to guide national health policy for PWH.

Table. Patient Characteristics

Poster: HERE

Kucher A, Clearfield E, Curtis RG, Delaney M, Germini F, Iorio A, O’Mahony B, Page D, Rotellini D, Skinner MW. Development of a refined data dashboard for PROBE: enhancing data interpretation and advocacy support for national patient organizations. Haemophilia. 2026;32:4-224. Abstract FP-038 (514). doi:10.1111/hae.70232.

Introduction

The PROBE (Patient-Reported Outcomes, Burdens and Experiences) study has collected standardized quality-of-life (QoL) data from people with hemophilia (PWH) and without a bleeding disorder since 2014, supported by a dashboard available since 2016. The dashboard allows the visualization of aggregated data. As more patient organizations participate in semi-annual data collection, there is an increasing need for more flexible, intuitive, and visually rich tools to interpret PROBE data and to support local reporting, advocacy, and scientific communications.

Aim is to develop and implement a new interactive dashboard using Microsoft Power BI that (1) improves visualization and interpretation of PROBE QoL data, (2) facilitates preparation of abstracts, reports, and presentations on progress and gaps within national hemophilia communities, and (3) provides a foundation for future PROBE modules for women with bleeding disorders, von Willibrand disease, and other rare bleeding and platelet disorders.

 Methods 

We redesigned the existing PROBE dashboard using Power BI, drawing on feedback from NMOs, clinicians, and patient partners. The dashboard, updated monthly, connects to the central PROBE database. Key PROBE metrics, including EQ-5D-5L, are organized into themed pages: Demographics; Mobility and Activities of Daily Living (ADL); Pain (Chronic and Acute); Employment and Education; Surgery and Co-morbid Conditions; Severity and Inhibitor Status; Treatment; Joints; and PROBE and EQ-5D-5L utility scores. Sections allow users to filter and stratify data (e.g., by age, severity, inhibitor status, country, or collection period) and to export figures and summary tables.

 Results

The PROBE dashboard provides interactive charts and tables that allow users to quickly identify patterns in QoL, including mobility limitations, pain burden, employment and education status, treatment patterns, and joint outcomes. Deep-dive views within each theme support more detailed discussions (e.g., comparing outcomes by severity or inhibitor status) and help patient organizations generate data-driven messages for advocacy and clinical quality improvement. The ability to export standardized visuals and summary statistics simplifies the preparation of national or multi-country reports describing QoL trends and progress, that could be used for research and advocacy.

 Conclusion 

The PROBE dashboard offers patient organizations an enhanced, user-friendly way to interpret QoL data. By improving access to clear, interpretable PROBE and EQ-5D-5L indicators, the dashboard supports global efforts to monitor outcomes, inform policy, and strengthen patient-centered care worldwide.

Presentation: HERE

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