Changing paradigm of prophylaxis with longer acting factor concentrates
Bibliographic record
Abstract
Beginning in the 1960s the care of persons with haemophilia began to improve dramatically through a series of transformative improvements in care: development of lyophilized factor concentrates, home care programmes, prophylaxis and (due to the tragedy of HIV/hepatitis) the development of virally safer plasma-derived and recombinant factor concentrates. Prophylaxis, if commenced early and given in sufficient dose/frequency has been shown to allow persons with haemophilia to maintain excellent joints and lead normal lives. Yet the relatively short half-lives of factor (F) VIII and IX concentrates leads to the need for frequent venous access. This remains a significant burden for patients with haemophilia on prophylaxis causing in many cases reduced patient adherence to prophylaxis and negative longterm outcomes. The last 5 years have witnessed a flourish of new bioengineered longer acting FVIII and IX concentrates manufactured using different technologies (pegylation or fusion to Fc/albumin). These products (especially the longer acting FIX concentrates) are likely to have profound implications on prophylaxis. With these longer acting factor concentrates prophylaxis regimens will almost certainly change. This will involve changes in what trough levels are targeted and how frequently factor is administered. It is hoped that these changes may improve patients' adherence to prophylaxis and their quality of life. These long-acting factor concentrates will undoubtedly have cost repercussions and will raise important questions regarding how decisions about choosing one longer acting concentrate over another, and whether these products are interchangeable, are made. This article will review what changes may ensue with the advent of these new longer acting factor concentrates.
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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 | 0.007 | 0.006 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.001 | 0.004 |
| Scholarly communication | 0.004 | 0.006 |
| Open science | 0.002 | 0.002 |
| Research integrity | 0.004 | 0.008 |
| Insufficient payload (model declined to judge) | 0.004 | 0.002 |
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".