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
In depth6:46CCAI
paperi.ai
0:00 / 0:00
Lisa J. Young, Yong Chen, Jose M. Alvir, Tom Burke, Enrico Ferri Grazzi, Ian C. Winburn
What changes when bleeding events become more frequent? In this European cohort, both health-related quality of life and work productivity declined as annual bleeding events increased.
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
What changes when bleeding events become more frequent? In this European cohort, both health-related quality of life and work productivity declined as annual bleeding events increased. Haemophilia A is associated with a substantial healthcare burden, with the greatest burden carried by individuals with severe disease.
In 2014, estimated total annual per-patient expenditures across five European countries averaged two hundred thousand euros, with ninety-eight percent of the economic burden driven by drug expenditures. Real-world evidence is crucial for quantifying the substantial burden of disease and informing the future treatment of haemophilia A.
The analysis explored the impact of bleeding events on health-related quality of life and work productivity in Europe using a subset of adults with haemophilia A without inhibitors from the CHESS II retrospective study. Participants were treated with either replacement factor products or a monoclonal antibody and were not participating in a clinical trial at recruitment.
The analysis focused on males aged eighteen to sixty-four years with haemophilia A without inhibitors who were receiving replacement factor products or a monoclonal antibody. The outcomes were analysed using EuroQol’s EQ-5D-5L index, the EQ-VAS analogue scale, and WPAI:SHP percentage scores, stratified by zero, one, two, three to four, or at least five annual bleeding events.
Eligible physicians retrospectively enrolled, on average, seven to eight patients with haemophilia for a twelve-month period before the clinical consultation, or index date. Physicians completed an electronic case report form for the patients they had most recently consulted with.
Bleeding events in the last twelve months were recorded. Health-related quality of life was evaluated using the EQ-5D-5L questionnaire and EQ-VAS. The EQ-5D-5L comprises five domains: mobility, self-care, usual activities, pain or discomfort, and anxiety or depression.
For the EQ-5D-5L index, a score of one equates to perfect health, while decreasing numbers indicate incrementally worse health using a UK value set and weights. The EQ-VAS recorded each patient’s self-rated health from one hundred, the best health imaginable, to zero, the worst health imaginable.
Table one describes the three hundred and eighteen patients with haemophilia A, grouped by annual bleeding events, or ABs: from zero through one, two, three or four, and at least five. It reports age, race and ethnicity, country, and employment status; mean age ranges from thirty point zero years in the zero-AB group to thirty-six point six in the three-or-four group.
The table provides the baseline context for interpreting later analyses of health and work-related outcomes across bleeding-event categories. Of nine hundred eighteen males with haemophilia A in CHESS II, three hundred eighteen met the inclusion criteria and had health-related quality-of-life data available; the mean age was thirty-three point eight years, and ninety-six percent were White.
The mean number of annual bleeding events over the preceding twelve months was two point seven, with twenty percent reporting three or four events and seventeen percent reporting at least five. Mean EQ-5D-5L index scores were zero point nine two for zero events, zero point seven six for one or two, zero point seven one for three to four, and zero point five six for at least five events.
Figure one links annual bleeding events with health-related quality of life in haemophilia A. The mean EQ-5D-5L index score is zero point nine two for patients with no annual bleeds and zero point five six for those with at least five, while mean self-rated EQ-VAS scores are eighty-six point nine and sixty-one point two, respectively.
Across mobility, self-care, usual activities, pain or discomfort, and anxiety or depression, the chart also reports the proportion with no or slight problems, showing the burden across specific health dimensions. Mean EQ-5D-5L index scores declined as bleeding events increased: zero point nine two for zero events, zero point seven six for one or two, zero point seven one for three to four, and zero point five six for at least five.
The percentage of patients in the top two EQ-5D-5L domain levels also reflected reduced health-related quality of life with increasing annual bleeding events. For mobility, ninety-six percent of patients with no events reported no or slight problems, compared with seventy-two percent of those with three or four and sixty-five percent of those with at least five.
Among patients with no events versus at least five, no or slight problems were reported for self-care by ninety-four versus seventy-four percent, usual activities by ninety-six versus sixty-seven percent, pain or discomfort by ninety-four versus fifty-two percent, and anxiety or depression by ninety-two versus seventy-eight percent.
Figure two shows mean overall work productivity loss due to haemophilia A, grouped by annual bleeding events in the CHESS II study. Among the one hundred fifty-one participants assessed, the chart reports an overall mean loss of twenty-eight point two eight percent, with separate estimates for zero, one, two, three or four, and at least five bleeding events, each accompanied by standard-deviation error bars.
This matters because the study links bleeding burden with work productivity, highlighting an important impact beyond clinical symptoms. These findings came from a retrospective analysis of existing observational CHESS II data, including medical records reported by treating physicians and questionnaires completed by patients in select European countries.
Because the study was retrospective, cross-sectional, and physician- and patient-reported, selection bias, recall bias, and potential data-extraction errors cannot be excluded. The analysis included adults up to age sixty-four but did not include subgroup analysis by age group.
Across this sample, more annual bleeding events were associated with worse quality-of-life and productivity scores. The pattern is important, but the cross-sectional design cannot establish that bleeding caused those outcomes.
A derivative work by Paperi · AI-generated script, voice and captions
· pages and figures unaltered
Made with Paperi.
Drop in a research PDF — get a narrated video walkthrough like this one,
with highlights that follow the narration. Free to start.
Clare Primiero, Brigid Betz‐Stablein, Nathan Ascott, Brian D’Alessandro, Séraphin Gaborit, Paul Fricker, Abigail Goldsteen, Sandra González-Villà, Katie L. Lee, Sana Nazari, Hang Thanh Nguyen, Valsamis Ntouskos, Frederik Pahde, Balázs Pataki, Josep Quintana, Susana Puig, Gisele Gargantini Rezze, Rafael García, H. Peter Soyer, Josep Malvehy
A skin-scanning system can be good at judging one spot, yet still miss the person around it. This paper asks whether the whole body, medical history, and change over time can make that judgment safer.Skin-cancer AI can outperform dermatologists in carefully controlled experiments, but what happens when it sees the whole patient, unfamiliar lesions, and real clinical context? This protocol builds the data pipeline to find out.
Nisa Ayob, Roelof Petrus Burger, Monray D. Belelie, Ncobile Nkosi, Henno Havenga, Lizaan de Necker, Dirk Cilliers
A disease carried by freshwater snails can move when the weather moves. By tracing where the snails could live, researchers mapped places in South Africa where infection may already be hiding, and where it could spread next.What if the geography of a disease could be reconstructed from old snail records and climate data? This study maps where South Africa’s schistosomiasis-transmitting snails were historically most likely to thrive.
David M. Presby, Summer R. Jasinski, Emily R. Capodilupo
Stress does not necessarily end when the stressful moment ends. A wearable may be able to see the body remain changed long after a hard run or a tense period of work.Stress usually gets measured by asking questions, drawing blood, or using bulky clinical equipment. This study asks whether a wrist wearable can detect stressful events passively, simply by tracking heart rate and heart rate variability.