Cross‐sectional study evaluating the association of haemophilia‐related distress and clinically relevant outcomes

Abstract

AbstractIntroductionIn chronic diseases, disease‐related distress can impact disease outcomes. Distress and haemophilia‐related distress has been demonstrated in people with haemophilia (PwH). The association of haemophilia‐related distress on disease outcomes among PwH is unknown.AimTo study the association of haemophilia‐related distress with haemophilia specific outcomes, including adherence to prophylactic therapy, the presence of a target joint, healthcare utilization and work‐impairment.MethodsIn a cross‐sectional study, adults with haemophilia A or B were enrolled in a study to validate the haemophilia‐related distress questionnaire (HRDq). In this planned analysis, univariate and multivariate associations between the HRDq total score and disease outcomes were explored.ResultsThe 114 participants in this analysis were male, mostly with haemophilia A (92%) and severe disease (52%) with a median age of 31.9 years. On univariate analysis, HRDq total score (5‐point change) was associated with the presence of a target joint (P = .002), high healthcare utilization (P = .011), poor adherence (P = .033) and work‐impairment (P ≤ .001). After adjustment for age, race, severity and other potential confounders, adherence (aβ 0.33, 95% CI .17, .49) and work‐impairment (aβ 4.69, 95% CI 3.27‐6.1) remained statistically significantly associated with HRDq total score.ConclusionHaemophilia‐related distress is associated with poor adherence to factor prophylaxis and work‐impairment. The direction of the association (causation) is yet to be determined and requires future study.