Evaluating Outcome of Prophylaxis in Haemophilia: Objective and Self-reported Instruments Should Be Combined

Introduction: Routine outcome assessment of prophylaxis should use validated tools, while balancing comprehensiveness and burden. Collecting overlapping information should be avoided.

Aim: To assess correlations between different outcome assessment tools in haemophilia.

Methods: From an international cross-sectional study, data on objective outcome (Haemophilia Joint Health Score (HJHS 2.1, range 0-124), radiological Pettersson score) and self-reported joint bleeding, Haemophilia Activities List (HAL, range 100-0), health-related quality of life (SF-36, including five physical and five mental domain scores, range 100-0), and Utility (SF6D and EQ-5D, range 1.0-0) were extracted. Spearman's correlations were calculated: ≥0.8 very strong, 0.60-0.79 strong, 0.40-0.59 moderate.

Results: Ninety patients with severe haemophilia, on prophylaxis since median age 3.4 years, were evaluated at median 25.5 years (range 16.0-37.6). Objective outcome was favourable (median HJHS 2.1 6 points, Pettersson score 9 points). Self-reported outcome showed a median of 7 joint bleeds in 5 years, median HAL sum 96 points, high scores for physical domains of SF-36 (median 80-95) and high Utility values (median SF6D 0.87; EQ-5D 0.84). Physical examination (HJHS 2.1) showed strong correlation with radiological scores, moderate correlation with physical domains of the SF-36 and Utility, but no correlation with self-reported bleeding or limitations in activities (HAL). Bleeding was not associated with any other outcome parameter. The HAL was only correlated with the SF36 'Physical functioning' domain.

Conclusion: For the evaluation of patients on early prophylaxis, information on bleeding should be complemented by objective joint assessment as well as self-reported limitations in activities and quality of life.