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

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.

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.

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.

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.

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.