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

Johanna Wilhelmina Hoefnagels*, Liesbeth Hélène Schrijvers, the Haemophilia in the Netherlands 6 Steering Group, Frank W.G. Leebeek, Jeroen Eikenboom, Saskia E.M. Schols, Cees Smit, Roger E.G. Schutgens, Samantha C. Gouw, Kathelijn Fischer

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

7 Citations (Scopus)
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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.

Original languageEnglish
Pages (from-to)581-590
Number of pages10
Issue number4
Publication statusPublished - 1 Jul 2021

Bibliographical note

Funding Information:
J.H.: has not declared any conflicts of interest. L.S.: has not declared any conflicts of interest. F.L. has received unrestricted research grants from CSL Behring, Takeda and uniQure, and is consultant for Takeda, uniQure, and Biomarin. He is DSMB member for a study sponsored by Roche. JE has received research support from CSL Behring and honorarium for educational activities of Roche and Celgene (all paid to the institute). SS has been paid for participation in an advisory board of NovoNordisk and she received a grant to participate in a masterclass of Takeda, both in 2020. CS has not declared any conflicts of interest. S.G. has received unrestricted research grants from Sobi. K.F. has received speaker's fees from Bayer, Baxter/Shire, Biotest, CSL Behring, Octapharma, Pfizer, NovoNordisk; performed consultancy for Baxter/Shire, Biogen, CSL Behring, Freeline, NovoNordisk, Pfizer, Roche and SOBI; and has received research support from Bayer, Pfizer, Baxter/Shire, and Novo Nordisk.

Publisher Copyright:
© 2021 UMC Utrecht. Haemophilia published by John Wiley & Sons Ltd.


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