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.

Keywords:

• health-related quality of life
• depression
• anxiety
• employment
• relationships

Acknowledgments

The authors acknowledge the medical writing assistance of Amy Ross, PhD, of ETHOS Health Communications in Yardley, Pennsylvania, which was supported financially by Novo Nordisk Inc., Plainsboro, New Jersey, in compliance with international Good Publication Practice guidelines. The abstract and select data from this paper were presented at the 2017 American Society of Hematology Conference as a poster presentation. The poster’s abstract was published in “Abstracts and Meeting Program” in Blood , 2017, 130:4730.

Abbreviations

AD, anxiety/depression; AP, average pain; BLE, basic lower extremity; B-HERO-S, Bridging Hemophilia B Experiences, Results and Opportunities into Solutions; BPI, Brief Pain Inventory v2 Short Form; CDC, Centers for Disease Control and Prevention; CLE, complex lower extremity; CP, current pain; GAD-7, Generalized Anxiety Disorder 7-Item; HAL, Hemophilia Activities List; HERO, Hemophilia Experiences, Results and Opportunities; HJHS, Hemophilia Joint Health Score; HRQoL, health-related quality of life; HTC, hemophilia treatment center; LP, least pain; MO, mobility; PD, pain/discomfort; P-FiQ, Pain, Functional Impairment, and Quality of Life; PHQ-9, Patient Health Questionnaire; PI, pain interference composite score; PRO, patient-reported outcome; PS, pain severity composite score; SC, self-care; SF-36, 36-Item Short Form Survey; UA, usual activities; UE, upper extremity; UDC, Universal Data Collection; VAS, visual analog scale; WP, worst pain.

Disclosure

T. Buckner has served on advisory boards with CSL Behring, Genentech, Novo Nordisk, Kedrion, Tremeau Pharmaceuticals, Bayer, Pfizer, Spark Therapeutics, and Shire and as a consultant for Uniqure. R. Sidonio has received grant support from Grifols/Kedrion, Genentech, Bioverativ (Sanofi) and Shire and has participated in advisory boards with Genentech, Shire, Biogen, CSL Behring, Aptevo, Bayer, Novo Nordisk, Octapharma, and Pfizer. M. Witkop has received grant funding from Pfizer, serves on advisory boards with Aptevo, Baxter Bioscience, Biogen Idec, Novo Nordisk, Octapharma, and Pfizer, and is on the Novo Nordisk Speakers Bureau. C. Guelcher has served on or is serving on nursing advisory boards with Biogen Idec, Baxter/Baxalta, Grifols, Novo Nordisk, Pfizer, and Octapharma and is on the Novo Nordisk Speakers Bureau and the Solution Sight Speakers Bureau. S. Cutter has received honoraria from Novo Nordisk and Pfizer. N. Iyer and D. Cooper are employees of Novo Nordisk Inc. The authors report no other conflicts of interest in this work.