Pain relief with Eptacog beta in haemophilia patients with inhibitors
Bibliographic record
Abstract
Introduction Bleeding episodes (BEs) into joints and muscles cause acute pain that can be reduced by quick and efficient treatment. Eptacog beta is a new recombinant coagulation factor VIIa approved by the EMA for the treatment of BEs in patients (≥12 years of age) with congenital hemophilia A or B with inhibitors (CHABwI) and for the prevention of bleeding in those undergoing surgery or invasive procedures. We herewith describe the impact of eptacog beta treatment on pain relief across two different age groups using data from the pivotal clinical trials. Method Eptacog beta pain score data originated from two pivotal Phase 3 clinical trials of similar design that evaluated the efficacy and safety of eptacog beta on BE in CHABwI, including one (PERSEPT1) conducted in adults/adolescents and the other one (PERSEPT2) in children. Subjects received initial doses of eptacog beta 75 or 225μg/kg followed by 75μg/kg dose at predefined intervals determined by clinical response. Pain was assessed by the subject, guardian, or caregiver using a visual analog scale (VAS). Successful pain relief was defined by a VAS pain score at 12 hours after eptacog beta administration being lower to that recorded prior to eptacog beta administration. Descriptive statistics were applied for pain analysis.[ 1 ] [ 2 ] Results Of the 52 subjects evaluated, 27 were ≥12 years old with a mean (±SD) age of 31.3 (±12.35) years and 25 <12 years old with a mean (±SD) age of 4.9 (±3.28) years. Majority of the 1017 treated BEs were successfully treated at 12 hours and at 24 hours in both age groups. The proportion of BEs with successful pain relief was approximately 90% in both groups, irrespective of BE severity. From baseline until 24 hours after eptacog beta administration, the VAS pain score consistently decreased at all time points in both age groups: mean (±SD) VAS pain score of 40.8 (±25.6) at baseline to 2.8 (±8.2) at 24 hours in the ≥12 year-group and from 50.6 (±25.1) to 6.7 (±14.9) in the <12 year-group, respectively. The median time to pain relief was 3 hours in the older group and above 5 hours in the younger. Concomitant analgesic requirement was less common among older vs younger patients (29.6% vs 48%, respectively) ([ Fig. 1 ]). Fig. 1 Evolution of pain VAS score by time points. Conclusion Eptacog beta resulted in clinically relevant pain reduction and bleed resolution across all age groups in CHABwI. A higher analgesic requirement and longer time to pain relief were observed in younger patients, possibly due to difficulty in assessing pain in this younger patient population. Publication History Article published online: 20 February 2023 © 2023. Thieme. All rights reserved. Georg Thieme Verlag Rüdigerstraße 14, 70469 Stuttgart, Germany
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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) | 0.001 | 0.001 |
| 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) | 0.003 | 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".