PB0666 Clinical Outcomes Following Treatment with Octocog Alfa in Patients with Hemophilia A: 9-Year Interim Read-Out of the Real-World AHEAD International Study
Bibliographic record
Abstract
without inhibitors.In children, median age at initiation of emicizumab was 3.2 (0,5-10) years, and three patients were younger than one year when they started emicizumab.Regarding children without inhibitors, one was an untreated (PUP) and 2 were minimally treated (MTP).Median follow-up time on emicizumab was 40 months (6-60).In a total cumulative time of 611 months, 9 patients (60%) had no treated bleeding events and, both the 2 MTP and the PUP did not develop breakthrough bleedings.Twelve bleeding events (in 6 patients) were confirmed and treated.These bleedings were mostly related to trauma or surgery (hemarthrosis; mucocutaneous hemorrhage; muscle hematomas and head trauma).In 5 episodes of head trauma (in 2 children), treatment was decided for bleeding prevention, but none of the patients had confirmed intracranial hemorrhage.Three patients experienced spontaneous hemarthrosis.Although target joints were previously reported in 6 patients, after this follow-up period no target joint was identified.Two major orthopedic surgeries and two minor procedures were performed under replacement therapy without any complications.No adverse events were observed.Conclusion(s): Although we report a small cohort and a very heterogeneous population, our results show an unexpectedly low bleeding rate in this high risk group.Invasive procedures, including 2 major orthopedic surgeries, were uneventful.
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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 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.000 | 0.001 |
| Research integrity | 0.001 | 0.001 |
| 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".