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DOI: 10.1055/s-0044-1785524
Disease Burden, Clinical Outcomes, and Quality of Life in People with Hemophilia A without Inhibitors in Europe: Analyses from CHESS II/CHESS PAEDs
Authors
Funding This analysis was funded by F. Hoffmann-La Roche Ltd. Medical writing support for the development of this manuscript, under the direction of the authors, was provided by Andrew Briggs, BA, and Rhiannon Owen, BSc, of Ashfield MedComms, an Inizio company, and funded by F. Hoffmann-La Roche Ltd.
Abstract
Introduction Limited data relating to treatment burden, quality of life, and mental health burden of hemophilia A (HA) are currently available.
Aim To provide a comprehensive overview of unmet needs in people with HA (PwHA) using data generated from the Cost of Haemophilia in Europe: a Socioeconomic Survey-II (CHESS II) and CHESS in the pediatric population (CHESS PAEDs) studies.
Methods CHESS II and CHESS PAEDs are cross-sectional surveys of European males with HA or hemophilia B (HB) aged ≥18 and ≤17 years, respectively. Participants with FVIII inhibitors, mild HA, or HB were excluded from this analysis, plus those aged 18 to 19 years. Annualized bleeding rates (ABRs), target joints, and other patient-reported outcomes were evaluated.
Results Overall, 468 and 691 PwHA with available data for the outcomes of interest were stratified by hemophilia severity and treatment regimen in CHESS II and CHESS PAEDs, respectively. In these studies, 173 (37.0%) and 468 (67.7%) participants received FVIII prophylaxis, respectively; no participants received the FVIII mimetic emicizumab or gene therapy. ABRs of 2.38 to 4.88 were reported across disease severity and treatment subgroups in both studies. Target joints were present in 35.7 and 16.6% of participants in CHESS II and CHESS PAEDS; 43.8 and 23.0% had problem joints. Chronic pain was reported by a large proportion of PwHA (73.9% in CHESS II; 58.8% in CHESS PAEDs). Participants also reported low EQ-5D scores (compared with people without HA), anxiety, depression, and negative impacts on their lifestyles due to HA.
Conclusions These analyses suggest significant physical, social, and mental burdens of HA, irrespective of disease severity. Optimization of prophylactic treatment could help reduce the burden of HA on patients.
Data Availability Statement
The data that support the findings of this study can be requested via the corresponding author and may be granted upon reasonable request. The data are not publicly available due to privacy and/or ethical restrictions.
Author Contributions
E.F.G., F.N., T.B., J.O.H., K.M., M.A., R.O. made substantial contributions to the conception and design of the work. B.N., E.F.G., F.N., K.K., M.A., P.C., R.O., T.B. contributed to the analysis and interpretation of data for the work. All authors revised the manuscript critically and provided final approval of the version to be published. All authors agree to be accountable for all aspects of the work.
Publication History
Received: 22 November 2023
Accepted: 04 March 2024
Article published online:
15 April 2024
© 2024. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution License, permitting unrestricted use, distribution, and reproduction so long as the original work is properly cited. (https://creativecommons.org/licenses/by/4.0/)
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