European retrospective study of real‐life haemophilia treatment
Bibliographic record
Abstract
Introduction Haemophilia treatment varies significantly between individuals, countries and regions and details of bleed rates, factor consumption and injection frequency are often not available. Aim To provide an overview of the FVIII / FIX treatment practice and outcome for patients with haemophilia A ( HA ) or haemophilia B ( HB ) across Europe. Methods Non‐interventional, 12‐month retrospective study where anonymized data were retrieved from haemophilia centres/registers in Belgium, France, Germany, Italy, Spain, Sweden and the United Kingdom. Male patients (all ages) receiving coagulation factor treatment 24 months prior to the study, with basal FVIII / FIX levels ≤5 IU dL −1 , without inhibitors, were included. Data were summarized descriptively. Results In total, 1346 patients with HA and 312 with HB were included in the analysis; 75% and 57% had severe disease ( FVIII / FIX < 1 IU dL −1 ) respectively. Prophylaxis was most common for severe haemophilia, especially for children, whereas on‐demand treatment was more common for moderate haemophilia in most countries. The mean ( SD ) prescribed prophylactic treatment ranged from 67.9 (30.4) to 108.4 (78.1) ( HA ) and 32.3 (10.2) to 97.7 (32.1) ( HB ) IU kg −1 per week, across countries. Most patients on prophylaxis were treated ≥3 times/week ( HA ) or two times/week ( HB ). The median annual bleeding rate ( ABR ) for patients on prophylaxis ranged from 1.0 to 4.0 for severe HA , and from 1.0 to 6.0 for severe HB , while those with moderate haemophilia generally had slightly higher ABR s. Median ABR s for on‐demand‐treated severe HA ranged from 4.5 to 18.0, and for HB , 1.5 to 14.0. Conclusion Treatment practice varied greatly between centres and countries and patients treated on‐demand and prophylactically both experienced bleeds, emphasizing the need for further optimization of care.
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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 | 0.000 | 0.000 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| 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) | 0.001 | 0.001 |
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".