Practice patterns in haemophilia A therapy – a survey of treatment centres in the United States
Bibliographic record
Abstract
A survey was conducted to ascertain practice patterns for haemophilia A therapy in the United States. Questionnaire data were supplied by 52 haemophilia centres with a total of 4129 patients under treatment. Most participating centres were affiliated with academic/teaching hospitals or institutions. Patients below 5 years comprised 17% of the study population, 6-18 years 41% and >18 years 42%, and the apportionment across severity categories was 53% severe, 17% moderate and 30% mild. Among patients with severe haemophilia, 49% were receiving on-demand treatment, while 44% were receiving some form of prophylaxis (13% primary, 20% secondary and 11% tertiary). Primary prophylaxis was the most common type in children below 5 years of age, accounting for 25% of this age group. In children 6-18 years old, 58% were on some type of prophylactic regimen, while on-demand treatment was most frequent among adults. Difficulties of venous access were the most frequently cited barrier to instituting prophylaxis. Catheters were in use among 37% of the patients under primary prophylaxis and 14% of those on secondary prophylaxis. No major differences were observed in forms of therapy used between larger and smaller centres. These observations provide an extensive characterization of haemophilia A practice patterns in the United States.
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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 | 0.002 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.001 | 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".