Perioperative Management with Efanesoctocog Alfa in Adults, Adolescents, and Children with Severe Hemophilia A in the Phase 3 XTEND Clinical Program
Bibliographic record
Abstract
Introduction: Efanesoctocog alfa (formerly BIVV001) is a first-in-class high-sustained factor VIII replacement therapy that provides effective bleed prevention with once-weekly dosing (50 IU/kg) in previously treated patients with severe hemophilia A. Here, we evaluate efficacy and safety of efanesoctocog alfa for perioperative management in the Phase 3 XTEND clinical program. Method: XTEND-1 (NCT04161495) and XTEND-Kids (NCT04759131) studies assessed safety and efficacy of efanesoctocog alfa in previously treated patients≥12 and<12 years old, respectively, with severe hemophilia A. Participants from each study could continue treatment in the XTEND-ed study (NCT04644575). Participants provided informed consent, and studies were approved by ethics committees. Participants undergoing surgery were to receive a pre-operative loading dose of efanesoctocog alfa 50 IU/kg. For major surgeries, post-operative doses of 30 or 50 IU/kg every 2–3 days were allowed. Surgery endpoints included dose, number of injections, hemostatic response, factor consumption, blood loss, and blood transfusions during the perioperative period. Interim data cut: January 17, 2023. Results: Forty-one participants (<18 years, n=9;≥18 years, n=32) underwent 49 major surgeries ([ Fig. 1 ]); 23 were orthopedic surgeries, with knee arthroplasty most common (n=12). All single 50 IU/kg pre-operative doses maintained hemostasis during major surgery. Median (range) number of doses per major surgery was 4.0 (1–7) during the perioperative period (Days−1 to 14). Hemostatic response was rated excellent for 43 surgeries and good for 5 surgeries. Median (range) blood loss was 20 (0–1000) mL during surgery (n=29); red blood cell transfusion was required for 1 total knee replacement. Of 32 minor surgeries ([ Fig. 2 ]) among 28 participants, 27 were managed with 1 pre-operative dose and 5 with no pre-operative dose. Of those with an assessment (n=25), all hemostatic responses were rated excellent. Fig. 1
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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 | 0.000 | 0.001 |
| 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".