Trial reportHaemophilia : the official journal of the World Federation of Hemophilia2022
Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors.
Trial report in Haemophilia : the official journal of the World Federation of Hemophilia, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
10 citing papers in PubMed, 14 citations in OpenAlex.
- Safety and Use of Eptacog Beta 225 µg/kg in Patients With Haemophilia A or B With Inhibitors.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Trial
- Bleed treatment with eptacog beta (rFVIIa) results in a low incidence of rebleeding in adult and adolescent patients with haemophilia A or B with inhibitors.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Trial
- Model-informed pediatric dose selection of marzeptacog alfa (activated): An exposure matching strategy.CPT: pharmacometrics & systems pharmacology · 2023Trial
- Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Trial
- Safety and real-world effectiveness of eptacog beta with emicizumab prophylaxis: an interim analysis of the ATHN 16 study.Blood vessels, thrombosis & hemostasis · 2026Article
- Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials.Blood vessels, thrombosis & hemostasis · 2025Article
- Transforming Hemophilia A Care: Insights into New Therapeutic Options.Life (Basel, Switzerland) · 2024Review
- Current status and future prospects of activated recombinant coagulation factor VIIa, NovoSeven®, in the treatment of haemophilia and rare bleeding disorders.Annals of hematology · 2024Review
- [Recent advances in the replacement therapy for Hemophilia].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2023Article
- [Chinese guidelines on the diagnosis and management of hemophilia with inhibitors (2023)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
31 authors at 20 institutions in 10 countries.
Funding
Abstract
introductionEptacog beta is a new recombinant activated human factor VII bypassing agent approved in the United States for the treatment and control of bleeding in patients with haemophilia A or B with inhibitors 12 years of age or older.
aimTo prospectively assess in a phase 3 clinical trial (PERSEPT 2) eptacog beta efficacy and safety for treatment of bleeding in children <12 years of age with haemophilia A or B with inhibitors.
methodsUsing a randomised crossover design, subjects received initial doses of 75 or 225 μg/kg eptacog beta followed by 75 μg/kg dosing at predefined intervals (as determined by clinical response) to treat bleeding episodes (BEs). Treatment success criteria included a haemostasis evaluation of 'excellent' or 'good' without use of additional eptacog beta, alternative haemostatic agent or blood product, and no increase in pain following the first 'excellent' or 'good' assessment.
resultsTreatment success proportions in 25 subjects (1-11 years) who experienced 546 mild or moderate BEs were 65% in the 75 μg/kg initial dose regimen (IDR) and 60% in the 225 μg/kg IDR 12 h following initial eptacog beta infusion. By 24 h, the treatment success proportions were 97% for the 75 μg/kg IDR and 98% for the 225 μg/kg IDR. No thrombotic events, allergic reactions, neutralising antibodies or treatment-related adverse events were reported.
conclusionBoth 75 and 225 μg/kg eptacog beta IDRs provided safe and effective treatment and control of bleeding in children <12 years of age.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.