Real-world utilities and health-related quality-of-life data in hemophilia patients in France and the United Kingdom
Received 24 January 2019
Accepted for publication 2 May 2019
Published 14 June 2019 Volume 2019:13 Pages 941—957
Checked for plagiarism Yes
Review by Single anonymous peer review
Peer reviewer comments 4
Editor who approved publication: Dr Johnny Chen
Liz Carroll,1 Gary Benson,2 Jérémy Lambert,3 Khadra Benmedjahed,3 Marek Zak,4 Xin Ying Lee5
1Haemophilia Society, London, UK; 2Northern Ireland Haemophilia Comprehensive Care Centre and Thrombosis Unit, Belfast City Hospital, Belfast, UK; 3Patient-Centered Outcomes, Mapi, an ICON plc company, Lyon, France; 4Global Development, Medical & Science, Biopharm, Novo Nordisk, Søborg, Denmark; 5Global Biopharm Patient Access, Novo Nordisk, Søborg, Denmark
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.
Keywords: quality-of-life, health utility, hemophilia A, hemophilia B, survey
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