A survey of the outcome of prophylaxis, on‐demand treatment or combined treatment in 18–35‐year old men with severe haemophilia in six countries
Bibliographic record
Abstract
A number of studies have been published on the benefits of prophylactic treatment in adults with haemophilia. However, in many countries, it is considered as optional due to financial constraints. This survey was carried out to examine the long-term effects of prophylaxis and the continuing benefit of the treatment into adulthood. Self-assessed health-related data and the EQ-5D questionnaire measuring health utility were collected from 124 men (26.9 ± 4.6 years) from Canada (N = 40), France (N = 14), Ireland (N = 17), the Netherlands (N = 16), Poland (N = 24) and the UK (N = 13). The respondents were split into four groups: On-Demand, <50% life on prophylaxis, ≥ 50% life on prophylaxis, Prophylaxis. Overall, long-term prophylaxis results in lower presence of target joints (P ≤ 0.001), occurrence of serious bleeding episodes (P ≤ 0.05), recurring bleeding episodes (P ≤ 0.01) and requirement for surgical procedures (P ≤ 0.05). Furthermore, health utility (P ≤ 0.01) in the On-demand group was significantly lower (P ≤ 0.01) compared to the ≥ 50% life on prophylaxis and the Prophylaxis group. No significant differences between countries were found except between the Netherlands and Poland, with Poland showing the lowest health utility (P ≤ 0.01) and the most problems with mobility (P ≤ 0.05) and pain/discomfort (P ≤ 0.001). The Netherlands showed the highest health utility (0.915) followed by Canada (0.791), Ireland (0.786), UK (0.768), France (0.687) and Poland (0.629). The results demonstrate consistently higher quality of life of individuals who are on long-term prophylactic treatment when compared to on-demand treatment or intermittent prophylaxis and on -demand treatment.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.000 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".