2024/5
Bibliographic record
Abstract
Background: Emicizumab reduces bleeding episodes in children with hemophilia A. This study aimed to better elucidate the effectiveness and safety of emicizumab prophylaxis in younger children with hemophilia A in South China. Methods: This multicenter observational study retrospectively analyzed data from children with hemophilia A with and without inhibitors treated with prophylactic emicizumab (June 2019 to May 2025). Individualized dosing was used. Bleeding rates were calculated from 183 days before (or from birth) to at least 6 months after switching to emicizumab. In the maintenance period, factor VIII was determined using bovine factor IXa and factor X in the factor VIII chromogenic Bethesda assay. Canadian Haemophilia Outcomes-Kids’ Life Assessment Tool score was used to assess health-related quality of life. Results: Among 26 patients (mean age 3.3 years), bleeding events at emicizumab initiation were reported in 16 patients (61.5%) during emicizumab treatment; most were trauma related. Bleeding events were significantly reduced after initiating or switching to emicizumab. The median every-7-day equivalent emicizumab maintenance dose was 0.81 mg/kg. The mean equivalent factor VIII level was 8.21% ± 3.74%, which positively correlated with emicizumab, independent of inhibitors. Comparable equivalent factor VIII levels and weekly emicizumab consumption were observed among patients with and without inhibitors after switching to emicizumab. Health-related quality of life improved after emicizumab (scores increased from 57.23 ± 16.63 to 69.79 ± 13.32). Safety was favorable, with no adverse events. Conclusions: While exploratory, the findings suggest low-dose emicizumab prophylaxis may be beneficial in young Chinese children with hemophilia A.
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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) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.002 | 0.001 |
| Science and technology studies | 0.001 | 0.001 |
| Scholarly communication | 0.004 | 0.002 |
| Open science | 0.002 | 0.003 |
| Research integrity | 0.003 | 0.001 |
| Insufficient payload (model declined to judge) | 0.809 | 0.676 |
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".