Treatment preferences in people with haemophilia A or caregivers of people with haemophilia A: A discrete choice experiment
Bibliographic record
Abstract
INTRODUCTION: The standard of care for people with haemophilia A (PwHA) is regular prophylactic therapy with Factor VIII. However, very little is known about the treatment preferences of PwHA with and without inhibitors, or their caregivers, when presented with the choice between intravenous and subcutaneous prophylactic treatments with different modes of action, side effect risk and frequency of administration. METHODS: We conducted a discrete choice experiment to assess the preferences of PwHA or their caregivers. The survey was conducted in Australia, New Zealand, Canada, France, Italy and the United Kingdom. RESULTS: There were 56 respondents. The majority of PwHA had hereditary HA (78%) rather than spontaneous HA (22%). Most PwHA were diagnosed when they were under 12 months old (65%). The model identified two segments based on treatment preferences. There is heterogeneity between PwHA in their treatment preferences. People with and without inhibitors prefer to receive treatment via a subcutaneous injection compared to an intravenous infusion. However, for PwHA, efficacy is key; they value a reduction in the annual bleed rate. For those without inhibitors, this also includes a reduction in the risk of developing inhibitors. The side effect risk, administration risk and storage requirements were prioritised differently in each segment. CONCLUSIONS: Results from this study may inform decisions about the value of existing and new treatments for PwHA.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.013 | 0.021 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.001 | 0.002 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.001 | 0.001 |
| Scholarly communication | 0.002 | 0.001 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.002 | 0.002 |
| Insufficient payload (model declined to judge) | 0.010 | 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 source (direct Gemma or distilled Codex), 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".