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The impact of bleeding event frequency on health-related quality of life and work productivity outcomes in a European cohort of adults with haemophilia A: insights from the CHESS II study

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Lisa J. Young, Yong Chen, Jose M. Alvir, Tom Burke, Enrico Ferri Grazzi, Ian C. Winburn

A bleeding event is not only a medical problem. In adults with haemophilia A, more bleeding was linked with poorer daily life and a much larger loss of work productivity.

Abstract

Background Haemophilia A carries a substantial healthcare burden, affecting health-related quality of life (HRQoL). The Cost of Haemophilia in Men: a Socioeconomic Survey II (CHESS II), a retrospective real-world study, characterised the burden of haemophilia and its impact on HRQoL and work productivity. The current analysis explored the impact of bleeding events on HRQoL and work productivity in Europe. This analysis focused on data collected from males aged 18 to 64 years with haemophilia A without inhibitors who were receiving replacement factor products or a monoclonal antibody and were not participating in a clinical trial at the time of study recruitment. Descriptive statistics were analysed using scores from EuroQoL’s EQ-5D-5L index and EQ-VAS analogue scale and the Work Productivity and Activity Index Specific Health Problem (WPAI:SHP) percentage scores stratified by the number of annual bleeding events (ABs) 0, 1, 2, 3–4, or ≥ 5. Results Of 918 males with haemophilia A in CHESS II, 318 met inclusion criteria and had data available for HRQoL measures; mean age (SD) was 33.8 (12.1) years and 96% were White. Mean (SD) ABs of 2.7 (2.9) occurred over the preceding 12 months: 20% had 3 or 4 ABs; 17% had ≥ 5 ABs. Mean EQ-5D-5L index scores for patients with 0, 1, 2, 3–4, or ≥ 5 ABs were 0.92, 0.76, 0.76, 0.71, and 0.56, respectively. Mean (SD) EQ-VAS scores were 86.9 (13.6), with 0 ABs versus 69.5 (19.1) for 3 or 4 ABs and 61.2 (17.2) for ≥ 5 ABs. Mean percentage of overall work productivity loss on the WPAI:SHP questionnaire ranged from 9.70 to 0 ABs to 47.65 for ≥ 5 ABs. Conclusions In this European sample of adult men with haemophilia A, HRQoL and work productivity scores were lower among those reporting more AB events. Bleeding burden appears to affect HRQoL and productivity; however, this cross-sectional analysis limits the ability to draw firm conclusions on causality.

Transcript

A bleeding event is not only a medical problem. In adults with haemophilia A, more bleeding was linked with poorer daily life and a much larger loss of work productivity. Haemophilia A is associated with a substantial healthcare burden, with the greatest burden carried by individuals with severe disease.

That burden is not only felt by patients and families. Across five European countries, the estimated yearly cost per patient with severe haemophilia A and B averaged nearly two hundred thousand euros, with most of the economic burden driven by drug expenditures. Real-world evidence is important for measuring the burden of haemophilia A and informing future treatment, especially as care continues to change.

To explore the impact of bleeding events on health-related quality of life and work productivity in Europe, the analysis examined adults with haemophilia A without inhibitors from an existing study. The analysis reviewed people receiving replacement factor products or a monoclonal antibody who were not taking part in a clinical trial when they joined the study.

Health-related quality of life was evaluated with questions about mobility, self-care, usual activities, pain or discomfort, and anxiety or depression. The overall health score treated one as perfect health, with lower numbers indicating worse health.

A separate question asked people to rate their own health from the best health they could imagine to the worst. They also reported more difficulty with movement, self-care, usual activities, pain, and mood. The mean overall health score declined as bleeding events increased, from 0.92 among people with no events to 0.56 among those with five or more events.

The same direction appeared across everyday concerns. People with more bleeding events were less likely to report no or slight problems with mobility, self-care, usual activities, pain or discomfort, and anxiety or depression. This links repeated bleeding to a substantial impact on working life.

These findings need careful interpretation because the analysis used existing observational data collected in the past from medical records and patient questionnaires. Because the study was retrospective, cross-sectional, and based on physician and patient reports, it cannot rule out selection and recall bias or errors in extracting data.

And because it analyzes existing observational data rather than assigning treatment, it cannot establish that bleeding itself caused every difference in quality of life or work productivity. The study found that severe haemophilia A was associated with reduced quality of life and reduced work productivity, and that both worsened with a greater number of bleeding events.

Treatments that reduce bleeding events may therefore bring improvements in quality of life and work productivity, offering renewed hope for people with haemophilia A. Across this European group, more bleeding went together with worse quality of life and less ability to work.

Reducing bleeding may therefore matter not only for health, but also for ordinary independence and working life.

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