Distress in people with hemophilia is associated with work impairment and poor adherence to factor prophylaxis, researchers reported. The conclusion was drawn from the analysis of a 4-domain hemophilia-related distress questionnaire (HRDq) composed of 24 questions assessing disease management, financial concerns, perceived self-efficacy, and daily function.

“Despite great advances in factor replacement therapy and the development of comprehensive [hemophilia treatment centers] in the past 40 years, this hemophilia-specific distress questionnaire suggests there are underlying psychological factors that hinder hemophilia disease management and negatively influence [people with hemophilia’s] behaviors, specifically adherence to prophylactic factor therapy,” the researchers wrote in Haemophilia.

HRDq total score was associated with work impairment and adherence to factor prophylaxis after adjusting for age, race, disease severity, and other potential confounders. For each 5-point increase in the HRDq total score, the researchers observed a 4.69% greater work impairment. The direction of the association, nevertheless, is yet to be determined.

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On bivariate analyses, the perceived self-efficacy score was associated with adherence to factor prophylaxis. Furthermore, a strong correlation was found between functional concerns and work impairment, whereas moderate correlations were found with hemophilia management concerns and perceived self-efficacy.

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In addition, HRDq total score and financial concerns and functional concerns scores were associated with the presence of a target joint bleeding on bivariate analysis. Moreover, the total score and perceived self-efficacy and functional concerns scores were associated with having a visit to the emergency department during the prior 6 months.

The functional concerns score was also associated with hospitalizations during the prior 6 months. However, these associations did not remain significant on multivariate analyses, after adjusting for covariates.

The study enrolled 114 male participants (median age, 31.9 years), mostly with hemophilia A (92%) and severe disease (52%).


Guasch S, Scott LC, Figueroa J, et al. Cross-sectional study evaluating the association of haemophilia-related distress and clinically relevant outcomes. Haemophilia. Published online January 14, 2023. doi:10.1111/hae.14741