Patient‐Reported Outcome Measures and Clinical Performance in Hemophilia: Implications for Person‐Centered Care

OBJECTIVES: To examine the association between patient-reported outcome measures (PROMs) and objective measures of clinical performance in children, adolescents, and adults with haemophilia, highlighting their implications for person-centered care. BACKGROUND: Advances in haemophilia treatment have reduced bleeding rates, increasing the relevance of patient-reported outcome measures (PROMs) to capture the real impact of the disease on a person's life. However, the integration of PROMs into clinical practice remains limited, despite the growing emphasis on person-centered, participatory, and preference-sensitive care. METHODS: Observational, cross-sectional, and analytical. 111 participants with haemophilia A (33 aged 8-17 years; 78 aged 18 years or older) were assessed at a Brazilian comprehensive haemophilia care centre. Patient-reported outcome assessment instruments (PROMs) included EQ-5D, VAS, PEDHAL, or HAL, depending on age. Clinical performance was assessed using the HJHS and FISH. Spearman correlations were applied. RESULTS: The PROMs showed moderate to strong correlations, clinically consistent with joint health and functional independence in both age groups. In children/adolescents, the PEDHAL showed a strong correlation with the HJHS. In adults, the HAL and EQ-5D-3L demonstrated the highest agreement with the HJHS and FISH. These findings indicate high convergent validity and reinforce patients' subjective perception as an accurate reflection of their functional status. CONCLUSIONS: The systematic use of these instruments can contribute to longitudinal monitoring, personalization of therapeutic strategies, and improvement in the quality of care, especially in adult populations that present a higher disease burden and a poorer perception of quality of life over time.