HaemoPREF: Further evaluation of patient perception and preference for treatment in a real world setting

Haemophilia. 2017 Nov;23(6):884-893. doi: 10.1111/hae.13321. Epub 2017 Sep 19.

Abstract

Introduction: Adherence to haemophilia A (HA) treatment may be influenced by patients' beliefs about their condition and treatment. Furthermore, difficulties administering treatment may lead to poor adherence. New treatment strategies aim to reduce the burden associated with administration and to improve patient perception of treatment, which, in turn, increase adherence levels.

Aims: This study aimed to examine patient perception of HA treatment and related factors using patient-reported outcome (PRO) questionnaires and to confirm the psychometric properties of a recently developed questionnaire, the HaemoPREF.

Methods: A non-interventional, cross-sectional, questionnaire study was conducted with adult HA patients in Spain (n=31), Germany (n=10) and Italy (n=48), who were using ReFacto AF with the FuseNGo administration device. Patients completed the HaemoPREF and other questionnaires measuring related constructs: treatment adherence, satisfaction and well-being, online at two time points. Correlational, regression and psychometric analyses were conducted.

Results: PRO scores indicated that patients are satisfied with and adherent to their treatment. Multivariate regression of the HaemoPREF global score identified a number of significant predictors (P≤.05). The HaemoPREF Global Score had a moderate relationship with subscales on the related questionnaires (mean correlation=0.43; range=0.39-0.48). The HaemoPREF demonstrated good test-retest reliability (intraclass correlation coefficient=0.82), internal consistency reliability (Cronbach's alpha range=0.69-0.82) and convergent validity with measures of treatment satisfaction (Spearman correlation coefficient, r=.48) and well-being (r=.41).

Conclusion: The findings suggest that patients using ReFacto AF with FuseNGo were satisfied with and adherent to their treatment. The HaemoPREF can identify important concepts relating to patient treatment experience in HA.

Keywords: Ease of Use; HaemoPREF; Haemophilia; Patient-Reported Outcome; Preference; Treatment Satisfaction.

Publication types

  • Multicenter Study

MeSH terms

  • Adult
  • Aged
  • Cross-Sectional Studies
  • Factor VIII / therapeutic use*
  • Germany
  • Hemophilia A / drug therapy*
  • Hemophilia A / psychology
  • Humans
  • Italy
  • Male
  • Middle Aged
  • Patient Preference / psychology
  • Patient Preference / statistics & numerical data*
  • Patient Satisfaction / statistics & numerical data*
  • Patients / psychology
  • Patients / statistics & numerical data*
  • Perception
  • Psychometrics / methods
  • Psychometrics / statistics & numerical data
  • Reproducibility of Results
  • Spain
  • Surveys and Questionnaires*
  • Treatment Adherence and Compliance / psychology
  • Treatment Adherence and Compliance / statistics & numerical data

Substances

  • Factor VIII