Adherence to prophylaxis and its association with activation of self‐management and treatment satisfaction

INTRODUCTION: Prophylactic replacement therapy (prophylaxis) in patients with haemophilia (PWH) requires lifelong, frequent (self)infusions. Prophylaxis effectiveness depends on adherence, and the drivers of treatment adherence among PWH are unclear. AIM: To quantify prophylaxis adherence and associations between adherence and patients’ treatment attitudes and satisfaction in a large cohort of children and adults with haemophilia. METHODS: In a nationwide, cross‐sectional, questionnaire‐based study, PWH with complete information currently using prophylaxis were selected. Validated Hemophilia Regimen Treatment Adherence Scale‐Prophylaxis (VERITAS‐Pro; normalised score range: 0–100, optimum 0) measured treatment adherence; the Patient Activation Measure (PAM‐13; total score range 0–100, optimum 100) measured activation of self‐management; Hemophilia Patient Satisfaction Scale (Hemo‐Sat; range 0–100, optimum 0) measured treatment satisfaction. Groups were compared according to age (children: <12 years; adolescents: 12–18 years; adults >18 years) and adherence levels using non‐parametric tests, and correlations were assessed using Spearman's rho. RESULTS: Among 321 participants (median age 33 years, interquartile range [IQR]:15–54 years), adherence was high (median VERITAS‐Pro total score 17, 89% adherent) but worsened with age, with median scores of 5, 14 and 20 in children, adolescents, adults, respectively (p < .001). Attitudes towards treatment (median 66 vs. 68) participants and treatment satisfaction (12 vs. 10) were similar between adherent and non‐adherent patients. The VERITAS‐Pro total score was moderately correlated with PAM‐13 (r = .41) but not with Hemo‐Sat (r = −.11). DISCUSSION: Prophylaxis adherence was high (89%) but decreased significantly with age and was not correlated with treatment attitude or treatment satisfaction.