Assessment of Quality of Life in Hemophilia Using PROMs: A Systematic Review

[EN] Objective: To identify and describe the patient-reported outcome measures (PROMs) most frequently used to assess quality of life (QoL) in individuals with hemophilia, based on a defned selection of recent scientifc literature. Method: A descriptive and exploratory systematic literature review was conducted following PRISMA guidelines. Searches were performed in PubMed, Scopus, Web of Science, CINAHL, and the Cochrane Library, targeting peer-reviewed articles published between January 1, 2013, and December 31, 2024. After applying the inclusion criteria, eight studies published between 2017 and 2023 were selected. The review included quantitative and qualitative studies assessing QoL in individuals with hemophilia using PROMs. The most frequently reported instruments were EQ-5D, Haem-A-QoL, CHO-KLAT, and Haemo-QoL. Studies focusing on other disorders, as well as reviews, editorials, letters, and clinical trials, were excluded. Data extraction focused on study characteristics, PROMs used, and reported outcomes. Methodological quality was assessed using the Joanna Briggs Institute (JBI) critical appraisal tools. Results: Of the 44 articles initially identifed, 8 met the inclusion criteria. EQ-5D was most commonly applied in adult populations, capturing multiple health dimensions, while Haem-A-QoL provided disease-specifc insights into health-related QoL. In pediatric populations, CHO-KLAT and Haemo-QoL were the most frequently used instruments, with CHO-KLAT undergoing revisions aimed at improving sensitivity to treatment-related burden. Considerable variability was observed in the selection and application of PROMs across studies and populations. Conclusion: PROMs, such as EQ-5D, Haem-A-QoL, CHO-KLAT, and Haemo-QoL, are widely used in the assessment of QoL in people with hemophilia and show practical applicability across age groups. Given the heterogeneity identifed, the fndings support the need to refne, adapt, and optimize existing instruments rather than implying comparative efectiveness. Greater harmonization in PROM use may improve comparability across studies and contribute to more consistent, patient-centered evaluation of QoL in hemophilia.