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CC BY 4.0 · TH Open 2025; 09: a26586151
DOI: 10.1055/a-2658-6151
Original Article

Mental Health, Work Productivity, and Quality of Life in People with Severe Haemophilia A Receiving Prophylaxis: Findings from the CHESS Data Platform

Authors

  • Pratima Chowdary 

    1   Department of Haematology, Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free London NHS Foundation Trust, Cancer Institute, University College London, London, United Kingdom

  • Letizia Polito

    2   Product Development and Strategy, F. Hoffmann-La Roche Ltd., Basel, Switzerland

  • Mark Oellerich

    2   Product Development and Strategy, F. Hoffmann-La Roche Ltd., Basel, Switzerland

  • Romain Chafaie

    2   Product Development and Strategy, F. Hoffmann-La Roche Ltd., Basel, Switzerland

  • Tom Burke

    3   Health Economics and Outcomes Research, HCD Economics, Daresbury, United Kingdom
    4   Department of Health and Social Care, University of Chester, Chester, United Kingdom

  • Tom Blenkiron 

    3   Health Economics and Outcomes Research, HCD Economics, Daresbury, United Kingdom

  • Enrico Ferri Grazzi 

    3   Health Economics and Outcomes Research, HCD Economics, Daresbury, United Kingdom

Funding This analysis of the CHESS study was funded by F. Hoffmann-La Roche, Ltd. The wider CHESS III–IV study was supported by unrestricted research grants from Roche and BioMarin.
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Abstract

Background

Newer therapeutic options for people with severe haemophilia A (PwSHA), in addition to improved clinical and patient-reported outcomes (PROs), have offered more personalised treatment regimens. This analysis explored mental health, work productivity, and health-related quality of life (HRQoL) among PwSHA in Europe receiving a prophylactic treatment regimen.

Methods

The Cost of Haemophilia: a Socio-economic Survey (CHESS) study is a retrospective cross-sectional study of adult men with haemophilia in Europe. We analysed data from CHESS participants with severe haemophilia A and no factor VIII (FVIII) inhibitors who received emicizumab or FVIII replacement prophylaxis. Data are from patient questionnaires and their treating health care providers. This analysis focused on PROs, including productivity and activity impairment via the Work Productivity and Activity Impairment, HRQoL via the EQ-5D-5L, and anxiety via the 7-item General Anxiety Disorder questionnaire (GAD-7) and depression via the 8-item Patient Health Questionnaire (PHQ-8). SHA treatment and clinical characteristics were also collected, including bleeding events, joint health, and chronic pain. All findings were analysed descriptively.

Results

A total of 350 PwSHA met the inclusion criteria, 94 (27%) of whom provided PROs. Most (68%; n = 64) were receiving emicizumab (FVIII prophylaxis, 32%; n = 30). Clinical characteristics were generally comparable between emicizumab and FVIII prophylaxis groups, including reported chronic pain (63% and 70%) and problem joints (61% and 63%), with on-demand FVIII use for the treatment of breakthrough bleeding events more commonly reported in the FVIII prophylaxis group (34% vs. 56%). Overall, HRQoL showed comparable EQ-5D-5L scores between the treatment groups, with a marginally higher score in the emicizumab group (0.71 vs. 0.69) compared with the FVIII prophylaxis group. Anxiety and depression scores were both numerically lower in the emicizumab group, suggesting a lower burden of disease (anxiety 7-item General Anxiety Disorder questionnaire [GAD-7] mean scores, 6.0 vs. 7.3; depression PHQ-8 mean scores, 6.8 vs. 7.8). Employed PwSHA in the emicizumab group reported a lower impact of SHA on their work impairment (31% vs. 50%), and only 19% (vs. 33%) of the emicizumab group required assistance with daily activities. More PwSHA receiving FVIII prophylaxis reported a negative impact of SHA on their ability to participate in social activities (70% vs. 56%) and on their physical activity (57% vs. 44%).

Conclusion

Patients receiving emicizumab prophylaxis appeared to have more favourable mental health, work productivity, and HRQoL-related outcomes than those receiving FVIII prophylaxis. These findings were observed in the context of comparable clinical characteristics between emicizumab and FVIII prophylaxis despite evidence of a more complex treatment history for the emicizumab group. This analysis has limitations, including a lack of adjustment for confounding factors.

Keywords

haemophilia A - severe haemophilia - emicizumab - mental health - work productivity - health-related quality of life - patient-reported outcomes - prophylaxis - burden of illness

Ethical Approval

The CHESS studies are approved by the Research Ethics Sub Committee of the Faculty of Health and Social care within the University of Chester, are conducted in collaboration with the UK Haemophilia Society and governed by a steering committee co-chaired by Mr. Brian O'Mahony, Chief Executive of the Irish Haemophilia Society and Mr. Randall Curtis, President of the Hemophilia Foundation of Northern California.


Supplementary Material



Publication History

Received: 03 March 2025

Accepted: 01 July 2025

Article published online:
04 August 2025

© 2025. 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/)

Georg Thieme Verlag KG
Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany

Bibliographical Record
Pratima Chowdary, Letizia Polito, Mark Oellerich, Romain Chafaie, Tom Burke, Tom Blenkiron, Enrico Ferri Grazzi. Mental Health, Work Productivity, and Quality of Life in People with Severe Haemophilia A Receiving Prophylaxis: Findings from the CHESS Data Platform. TH Open 2025; 09: a26586151.
DOI: 10.1055/a-2658-6151
 

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