Download PDF
CC BY 4.0 · TH Open 2024; 08(02): e181-e193
DOI: 10.1055/s-0044-1785524
Original Article

Disease Burden, Clinical Outcomes, and Quality of Life in People with Hemophilia A without Inhibitors in Europe: Analyses from CHESS II/CHESS PAEDs

Pratima Chowdary 
1   Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free London NHS Foundation Trust, London, United Kingdom
,
Richard Ofori-Asenso
2   Real-World Data Enabling Platform, Roche Products Ltd, Welwyn Garden City, United Kingdom
,
Francis Nissen
3   Department of Real-World Data, F. Hoffmann-La Roche Ltd, Basel, Switzerland
,
Enrico F. Grazzi
4   Health Economics and Outcomes Research, HCD Economics, Daresbury, United Kingdom
,
Martynas Aizenas
5   Department of Access Strategy, F. Hoffmann-La Roche Ltd, Basel, Switzerland
,
Katya Moreno
6   Department of Product Development and Medical Affairs, F. Hoffmann-La Roche Ltd, Basel, Switzerland
,
Tom Burke
4   Health Economics and Outcomes Research, HCD Economics, Daresbury, United Kingdom
7   Department of Health and Social Care, University of Chester, Chester, United Kingdom
,
Beatrice Nolan
8   Department of Haematology, Children's Health Ireland at Crumlin, Dublin, Ireland
,
Jamie O'Hara
4   Health Economics and Outcomes Research, HCD Economics, Daresbury, United Kingdom
7   Department of Health and Social Care, University of Chester, Chester, United Kingdom
,
Kate Khair
9   Department of Research, Haemnet, London, United Kingdom
› Author Affiliations 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.
› Further Information
   Permissions and Reprints

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.

Keywords

congenital hemophilia A - real-world data - disease burden

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.


Supplementary Material



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/)

Georg Thieme Verlag KG
Rüdigerstraße 14, 70469 Stuttgart, Germany

 

  • References

  • 1 Srivastava A, Santagostino E, Dougall A. et al. WFH guidelines for the management of haemophilia, 3rd ed. Haemophilia 2020; 26 (S6): 1-158
    CrossrefPubMedGoogle Scholar
  • 2 Nissen F, Burke T, Asghar S. et al. An insight into clinical outcomes in mild, moderate, and severe haemophilia A (HA): a preliminary analysis of the CHESS II study. Res Pract Thromb Haemost 2020;4(01):
    PubMedGoogle Scholar
  • 3 Castro F, Silkey M, Khair K. et al. Joint bleeds in paediatric patients with haemophilia A: a CHESS study analysis. Res Pract Thromb Haemost 2019;3(Suppl 1).
    PubMedGoogle Scholar
  • 4 Hanley J, McKernan A, Creagh MD. et al; Musculoskeletal Working Party of the UKHCDO. Guidelines for the management of acute joint bleeds and chronic synovitis in haemophilia: a United Kingdom Haemophilia Centre Doctors' Organisation (UKHCDO) guideline. Haemophilia 2017; 23 (04) 511-520
    CrossrefPubMedGoogle Scholar
  • 5 Tagliaferri A, Feola G, Molinari AC. et al; POTTER Study Group. Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study. Thromb Haemost 2015; 114 (01) 35-45
    PubMedGoogle Scholar
  • 6 O'Hara J, Walsh S, Camp C. et al. The impact of severe haemophilia and the presence of target joints on health-related quality-of-life. Health Qual Life Outcomes 2018; 16 (01) 84
    CrossrefPubMedGoogle Scholar
  • 7 O'Hara J, Walsh S, Camp C. et al. The relationship between target joints and direct resource use in severe haemophilia. Health Econ Rev 2018; 8 (01) 1
    CrossrefPubMedGoogle Scholar
  • 8 Berntorp E, Dolan G, Hay C. et al. European retrospective study of real-life haemophilia treatment. Haemophilia 2017; 23 (01) 105-114
    CrossrefPubMedGoogle Scholar
  • 9 Scott MJ, Xiang H, Hart DP. et al. Treatment regimens and outcomes in severe and moderate haemophilia A in the UK: the THUNDER study. Haemophilia 2019; 25 (02) 205-212
    CrossrefPubMedGoogle Scholar
  • 10 Batty P, Austin SK, Khair K. et al. Treatment burden, haemostatic strategies and real world inhibitor screening practice in non-severe haemophilia A. Br J Haematol 2017; 176 (05) 796-804
    CrossrefPubMedGoogle Scholar
  • 11 Kizilocak H, Young G. Diagnosis and treatment of hemophilia. Clin Adv Hematol Oncol 2019; 17 (06) 344-351
    PubMedGoogle Scholar
  • 12 Peyvandi F, Tavakkoli F, Frame D. et al. Burden of mild haemophilia A: systematic literature review. Haemophilia 2019; 25 (05) 755-763
    CrossrefPubMedGoogle Scholar
  • 13 Miesbach W, Kittler S, Bauhofer A. et al. Long-term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A. Haemophilia 2020; 26 (03) 467-477
    CrossrefPubMedGoogle Scholar
  • 14 Wells JR, Gater A, Marshall C, Tritton T, Vashi P, Kessabi S. Exploring the impact of infusion frequency in haemophilia A: exit interviews with patients participating in BAY 94–9027 extension studies (PROTECT VIII). Patient 2019; 12 (06) 611-619
    CrossrefPubMedGoogle Scholar
  • 15 Canaro M, Goranova-Marinova V, Berntorp E. The ageing patient with haemophilia. Eur J Haematol 2015; 94 (Suppl. 77) 17-22
    CrossrefPubMedGoogle Scholar
  • 16 Witkop M, Neff A, Buckner TW. et al. Self-reported prevalence, description and management of pain in adults with haemophilia: methods, demographics and results from the Pain, Functional Impairment, and Quality of life (P-FiQ) study. Haemophilia 2017; 23 (04) 556-565
    CrossrefPubMedGoogle Scholar
  • 17 Witkop ML, Lambing A, Nichols CD, Munn JE, Anderson TL, Tortella BJ. Interrelationship between depression, anxiety, pain, and treatment adherence in hemophilia: results from a US cross-sectional survey. Patient Prefer Adherence 2019; 13: 1577-1587
    CrossrefPubMedGoogle Scholar
  • 18 O'Hara J, Hughes D, Camp C, Burke T, Carroll L, Diego DG. The cost of severe haemophilia in Europe: the CHESS study. Orphanet J Rare Dis 2017; 12 (01) 106
    CrossrefPubMedGoogle Scholar
  • 19 EQ-5D-Y user guide: how to apply, score, and present results from the EQ-5D-Y. 2020 . Accessed October 31, 2023 at: https://euroqol.org/wp-content/uploads/2019/10/EQ-5D-Y-User-Guide.pdf
    PubMedGoogle Scholar
  • 20 van Hout B, Janssen MF, Feng YS. et al. Interim scoring for the EQ-5D-5L: mapping the EQ-5D-5L to EQ-5D-3L value sets. Value Health 2012; 15 (05) 708-715
    CrossrefPubMedGoogle Scholar
  • 21 Ramos-Goñi JM, Oppe M, Estévez-Carrillo A, Rivero-Arias O. IMPACT HTA HRQoL Group. Accounting for unobservable preference heterogeneity and evaluating alternative anchoring approaches to estimate country-specific EQ-5D-Y value sets: a case study using Spanish preference data. Value Health 2022; 25 (05) 835-843
    CrossrefPubMedGoogle Scholar
  • 22 Rabin R, de Charro F. EQ-5D: a measure of health status from the EuroQol group. Ann Med 2001; 33 (05) 337-343
    CrossrefPubMedGoogle Scholar
  • 23 McLaughlin P, Hermans C, Sohaib A. et al. Problem joints and their clinical and humanistic burden in children and adults with moderate and severe haemophilia A: CHESS Paediatrics and CHESS II. Blood 2020; 136 (Suppl. 01) 33-34
    CrossrefPubMedGoogle Scholar
  • 24 Al-Huniti A, Reyes Hernandez M, Ten Eyck P, Staber JM. Mental health disorders in haemophilia: systematic literature review and meta-analysis. Haemophilia 2020; 26 (03) 431-442
    CrossrefPubMedGoogle Scholar
  • 25 Grochtdreis T, Dams J, König HH, Konnopka A. Health-related quality of life measured with the EQ-5D-5L: estimation of normative index values based on a representative German population sample and value set. Eur J Health Econ 2019; 20 (06) 933-944
    CrossrefPubMedGoogle Scholar
  • 26 Hernandez G, Garin O, Pardo Y. et al. Validity of the EQ-5D-5L and reference norms for the Spanish population. Qual Life Res 2018; 27 (09) 2337-2348
    CrossrefPubMedGoogle Scholar
  • 27 Jensen MB, Jensen CE, Gudex C, Pedersen KM, Sørensen SS, Ehlers LH. Danish population health measured by the EQ-5D-5L. Scand J Public Health 2023; 51 (02) 241-249
    CrossrefPubMedGoogle Scholar
  • 28 Iannone M, Pennick L, Tom A. et al. Prevalence of depression in adults with haemophilia. Haemophilia 2012; 18 (06) 868-874
    CrossrefPubMedGoogle Scholar
  • 29 Skinner MW, Négrier C, Paz-Priel I. et al. The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies. Haemophilia 2021; 27 (05) 854-865
    CrossrefPubMedGoogle Scholar