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

Abstract

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.

Transition readiness among adolescents and young adults with haemophilia in the Netherlands: Nationwide questionnaire study

“…Both domain scores and overall VERITAS‐Pro scores are presented as a standardized, continuous variable ranging from 0 to 100, with lower scores indicating better adherence. Good treatment adherence is defined as a standardized total score below 34 25 . VERITAS‐Pro's reliability among Dutch boys with severe haemophilia using prophylaxis is good (Cronbach's α =0.70).…”

“…Good treatment adherence is defined as a standardized total score below 34. 25 VERITAS-Pro's reliability among Dutch boys with severe haemophilia using prophylaxis is good (Cronbach's α =0.70). Domain reliability varies from 0.39 ('Dose') to 0.73 ('Skip').…”

“…Yet, it is hypothesized that some patients are deliberately not strictly adherent to their prophylaxis schedule and adjust infusions according to their activities, like sports. 25 So, even though the VERITAS-Pro considers this 'low adherence' , it could be considered a sign of good treatment insight. Thus, VERITAS-Pro scores should be interpreted with caution.…”

Disability and the social impact of hemophilia

“…It is a period characterized by rebellion, which also involves rebelling against hemophilia and trying to find a culprit. This defiance may pose a risk for therapeutic adherence as taking the medication inevitably interferes with adolescents’ daily activities [6]. At this stage in life, patients must take greater responsibility for their treatment.…”

Colorectal Cancer Screening in Patients with Inherited Bleeding Disorders: High Cancer Detection Rate in Hemophilia Patients

“…The HiN6 study is the 6th nationwide, multicenter, cross-sectional study among patients with hemophilia in the Netherlands and was performed between May 2018 and August 2019 [23]. The recruitment methods have been described previously [24]. All male patients with severe, moderate, or mild hemophilia A or B, who were treated at a Hemophilia Treatment Center in the Netherlands, were eligible for inclusion in this study [23].…”