Correlations between patient-reported outcomes and self-reported characteristics in adults with hemophilia B and caregivers of children with hemophilia B: analysis of the B-HERO-S study

Abstract

Purpose: Pain, anxiety, depression, and other aspects of health-related quality of life (HRQoL) are important issues for people with hemophilia and caregivers of children with hemophilia. Patient-reported outcome (PRO) instruments may be used to assess aspects of HRQoL; however, the use of PROs in clinical management of patients with hemophilia is limited and inconsistent. The Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S) study evaluated the impact of hemophilia B on HRQoL and other psychosocial aspects in affected adults and caregivers of children with hemophilia B. This post hoc analysis assessed correlations between PRO scores and psychosocial questions commonly asked in comprehensive care settings among B-HERO-S respondents. Patients and methods: B-HERO-S consisted of two online surveys, one administered to adults with hemophilia B (n=299) and one administered to caregivers of children with hemophilia B (n=150). The adult survey included EQ-5D-5L with visual analog scale, BPI, HAL, and PHQ-9. The caregiver survey included PHQ-9 and GAD-7. Questions related to demographics, hemophilia treatment, and psychosocial questions asked in comprehensive care visits were also included in the surveys. A post hoc analysis was performed to assess correlations between responses to selected psychosocial questions with PRO scores. Results: For adults with hemophilia B, greater pain severity and pain interference scores were associated with work-related problems, functional limitations, and relationship, psychological , and treatment issues. Significant correlations were also noted between some of these psychosocial outcomes and depressive symptoms. For caregivers, greater depression and anxiety were associated with employment issues, their child's functional, relationship, and psychological issues, having had difficulty or concerns with treatment/factor availability or affordability, and having less frequent HTC visits. Conclusion: High correlations were observed between PRO scores measuring pain, depression , and anxiety and questions commonly used in the comprehensive care setting to assess the psychosocial impact of hemophilia.