Abstract
Purpose: Congenital hemophilia A and B are bleeding disorders characterized by deficiency of factors VIII and IX, respectively. This study aimed to collect health-related quality-of-life (HRQoL) and health-utility data from hemophilia patients with differing disease severity.
Methods: Individuals with hemophilia aged ≥12 years living in France or the UK completed a series of questionnaires, including the EQ-5D-3L and -5L and SF-36 version 2. Association with demographic and clinical variables was explored using linear regression, and health-utility comparison was completed using Pearson and intraclass correlation coefficients.
Results: A total of 122 patients in France and 62 in the UK completed the survey. The combined sample primarily consisted of hemophilia A patients, mean age of 41 years, 70% had severe hemophilia, and 56% were on long-term prophylaxis. Similar HRQoL and utility scores were observed across the French and UK samples. The presence of more than two target joints, occurrence of joint surgery, and increased joint-pain frequency were independent predictors of lower SF-36 — physical health summary scores and lower health-utility scores. No statistically significant reductions in SF-36 — mental health summary scores were observed, except for participants with target joints. Strong correlations were observed between health- utility values derived from the three instruments (r=0.69–0.79).
Conclusion: Results of this study reinforce the importance of appropriate treatment to limit the physical burden and long-term joint damage associated with hemophilia. Further, utility values collected here reflect real-world data, and can serve as health-state weights in future cost–utility analyses.
Acknowledgments
The authors would like to thank all participants in France and the UK. The authors would also like to thank Claire Arcé, Thomas Sannié and Blanche Debaecker (French Haemophilia Association - AFH), Anila Babla (The Haemophilia Society) and Emmanuelle Devanne (Mapi) for their logistic support, Christina Hoxer (Novo Nordisk) for the contribution in the study set up, Pia Rægaard Christoffersen (Novo Nordisk) for her input and critical review of the manuscript, Tan P Pham (Mapi) for his support in the statistical analyses and Selam Shah (Mapi) for her support and assistance in medical writing. This study was funded by Novo Nordisk.
Abbreviation list
HRQoL, health-related quality-of-life; PRO, patient-reported outcome; EQ-5D, EuroQol — 5 dimensions; SF-36, 36-item Short-Form Health Survey; PCS, physical component summary; MCS, mental component summary.
Data-sharing statement
Data sets generated during and/or analyzed during the current study are available from the corresponding author (XYL) on reasonable request.
Supplementary materials
Table S1 Health-utility values and health-related quality-of life-scores by country, hemophilia type, and treatment regimen in the subsample of subjects with severe hemophilia (n=130)
Table S2 Health-utility values and health-related quality-of-life scores by number of target joints, joint pain, and joint surgery occurrence in the subsample of subjects with severe hemophilia (n=130)
Table S3 Health-utility values and health-related quality-of-life scores by history of long hospital stays and frequency of visits to medical professionals due to hemophilia in subsample of subjects with severe hemophilia (n=130)
Author contributions
XYL, MZ, JL, and KB contributed to concept and design, analysis and interpretation of data; LC contributed to the data collection and the interpretation of data. GB contributed to the interpretation of the data. All authors contributed to data analysis, drafting and revising the article, gave final approval of the version to be published, and agree to be accountable for all aspects of the work.
Disclosure
JL is an employee of Mapi, an ICON plc company, was also a paid consultant to Novo Nordisk and Roche. KB is an employee of Mapi, an ICON plc company, was also a paid consultant to Novo Nordisk. LC is an employee of the Haemophilia Society, which has received financial compensation from Novo Nordisk. XYL and MZ are employees of Novo Nordisk . GB was part of the advisory board for Novo Nordisk and received speaker fees from Novo Nordisk, Bristol Myers Squibb, Shire and Bayer. LC reports financial compensation from Novo Nordisk, outside the submitted work. GB reports personal fees from Bristol Myers Squibb Bayer, Baxalta, Pfizer, personal fees and non-financial support from Sobi and Novo Nordisk, outside the submitted work. The authors report no other conflicts of interest in this work.