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      Patient Preference and Adherence (submit here)

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

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          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.

          Most cited references24

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          The estimation of a preference-based measure of health from the SF-36.

          This paper reports on the findings of a study to derive a preference-based measure of health from the SF-36 for use in economic evaluation. The SF-36 was revised into a six-dimensional health state classification called the SF-6D. A sample of 249 states defined by the SF-6D have been valued by a representative sample of 611 members of the UK general population, using standard gamble. Models are estimated for predicting health state valuations for all 18,000 states defined by the SF-6D. The econometric modelling had to cope with the hierarchical nature of the data and its skewed distribution. The recommended models have produced significant coefficients for levels of the SF-6D, which are robust across model specification. However, there are concerns with some inconsistent estimates and over prediction of the value of the poorest health states. These problems must be weighed against the rich descriptive ability of the SF-6D, and the potential application of these models to existing and future SF-36 data set.
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            • Record: found
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            Definitions in hemophilia: communication from the SSC of the ISTH.

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              • Abstract: found
              • Article: not found

              Deriving a preference-based single index from the UK SF-36 Health Survey.

              This article presents the results of a study to derive a preference-based single index from the SF-36. The study was an attempt to reconcile a profile health status measure, the SF-36, with the "quality adjusted life years" approach. The study undertook a parsimonious restructuring of the SF-36 using explicit criteria to form the SF-6D health state classification. A sample of multidimensional health states defined by this classification were valued by a convenience sample of health professionals, managers, and patients, who responded to a set of visual analogue scale ratings and standard gamble questions, with highly complete and consistent answers. Statistical models were estimated to predict single index scores for all 9000 health states defined by the new classification. The resultant algorithms can be applied to existing SF-36 data sets and used in the assessment of the cost-effectiveness of health technologies. This preliminary work forms the basis of a larger study currently being undertaken in the UK.
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                Author and article information

                Journal
                Patient Prefer Adherence
                Patient Prefer Adherence
                PPA
                ppa
                Patient preference and adherence
                Dove
                1177-889X
                14 June 2019
                2019
                : 13
                : 941-957
                Affiliations
                [1 ] Haemophilia Society , London, UK
                [2 ] Northern Ireland Haemophilia Comprehensive Care Centre and Thrombosis Unit, Belfast City Hospital , Belfast, UK
                [3 ] Patient-Centered Outcomes, Mapi, an ICON plc company , Lyon, France
                [4 ] Global Development, Medical & Science, Biopharm, Novo Nordisk , Søborg, Denmark
                [5 ] Global Biopharm Patient Access, Novo Nordisk , Søborg, Denmark
                Author notes
                Correspondence: Xin Ying Lee Global Biopharm Patient Access, Novo Nordisk , 108 Vandtårnsvej, Søborg, DenmarkTel +45 30 777 030 Email xlee@ 123456novonordisk.com
                Article
                202773
                10.2147/PPA.S202773
                6585419
                31354248
                50d86f02-a4a6-4f3c-8a62-52f191a501ed
                © 2019 Carroll et al.

                This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution – Non Commercial (unported, v3.0) License ( http://creativecommons.org/licenses/by-nc/3.0/). By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms ( https://www.dovepress.com/terms.php).

                History
                : 24 January 2019
                : 02 May 2019
                Page count
                Tables: 5, References: 35, Pages: 17
                Categories
                Original Research

                Medicine
                quality-of-life,health utility,hemophilia a,hemophilia b,survey
                Medicine
                quality-of-life, health utility, hemophilia a, hemophilia b, survey

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