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Real-world utilities and health-related quality-of-life data in hemophilia patients in France and the United Kingdom

Authors Carroll L, Benson G, Lambert J, Benmedjahed K, Zak M, Lee XY

Received 24 January 2019

Accepted for publication 2 May 2019

Published 14 June 2019 Volume 2019:13 Pages 941—957

DOI https://doi.org/10.2147/PPA.S202773

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Colin Mak

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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