OC 24.4 Effect of F8 Mutations and Von Willebrand Factor on Pharmacokinetics of FVIII Concentrates in Severe Hemophilia A
Bibliographic record
Abstract
bleeds in life, 10 (4%) with at least a spontaneous joint bleed.The mean annualized joint bleeding rate (AJBR) and spontaneous AJBR were 0.0155 [standard deviation (SD): 0.0317] and 0.0014 (SD: 0.0097), respectively.Lower mean FVIII levels were observed in patients having a history of joint bleeds (17.8 vs 26.8, p < 0.0001).After adjusting for age and blood group, with every IU/dL increase of FVIII level, we observed a 7% reduction of AJBR and a 34% reduction of spontaneous AJBR (Table 1).The minimum FVIII trough level needed to prevent any and spontaneous joint bleeds lifelong resulted to be 19.2IU/dL and 17.7 IU/dL, respectively (Table 2). Conclusion(s):In our large Italian cohort of mild HA patients, we identified the minimum target FVIII level to prevent total and spontaneous joint bleeds (19.2 and 17.7 IU/dL, respectively).These findings could contribute to identify the target level to be attained in the frame of optimal prophylaxis and gene therapy.
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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 | 0.001 | 0.003 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 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 | 0.000 |
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 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".