ArticleOrphanet journal of rare diseases2025
The association of factor VIII activity levels with bleeding and quality of life in haemophilia a: findings from the European CHESS II study.
Article in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- Normalization of Haemostasis in People with Haemophilia A: Expert Consensus on Unmet Needs and a Framework for Advancing Towards Health Equity.TH open : companion journal to thrombosis and haemostasis · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
Abstract
backgroundHaemophilia A is a rare disorder leading to excessive bleeding, resulting from a deficiency in circulating clotting factor VIII. While we know that raising factor VIII levels for extended periods of time would reduce the frequency of injections required and provide more consistent bleed protection, the relationship between factor activity levels (FALs), clinical outcomes, and health-related quality of life (HRQoL) for people with haemophilia A (PwHA) has not been well characterised.
objectivesThis study explored the relationship of FALs with annual bleeding rate (ABR) and HRQoL in PwHA.
methodsThis retrospective, cross-sectional study used data from the CHESS II study (2018-2020) including men aged ≥ 18 years with haemophilia A in Europe. Physicians provided patient characteristics and clinical outcomes from the medical records. Patients completed a questionnaire with HRQoL (EQ-5D) and socio-economic information. PwHA receiving on-demand treatment who had baseline factor VIII levels < 40 IU/dL and no inhibitors were included. Regression models explored FALs with ABR and HRQoL controlling for age, BMI, and presence of blood-borne viruses.
resultsA total of 403 PwHA were included (167 provided HRQoL responses). Mean age was 37 years. Mean baseline FAL was 7.0 IU/dL and mean ABR was 2.3 (SD, 2.64). A negative binomial model showed for every 1% increase in FVIII levels, ABR decreased by 3.9% (0.09 events; P < 0.001). A tobit model showed that every 1% increase in FVIII levels was associated with an increase of 0.0054 points in mean EQ-5D index score (P < 0.001).
conclusionsThis study offers tangible estimates of how higher FALs may relate to lower ABR and elevated HRQoL for PwHA.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.