ArticleHaemophilia : the official journal of the World Federation of Hemophilia2024
Real-world effectiveness of eptacog beta in patients with haemophilia and inhibitors: A multi-institutional case series.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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Who cites it
6 citing papers in PubMed.
- 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
- Safety and real-world effectiveness of eptacog beta with emicizumab prophylaxis: an interim analysis of the ATHN 16 study.Blood vessels, thrombosis & hemostasis · 2026Article
- Real-World Experience with Eptacog Beta for On-Label and Off-Label Indications: The Spanish Experience.Pharmaceuticals (Basel, Switzerland) · 2025Article
- Recombinant factor VIIa: new insights into the mechanism of action through product innovation.Research and practice in thrombosis and haemostasis · 2025Review
- Real-world effectiveness of eptacog beta in patients with haemophilia and inhibitors: A multi-institutional case series.Haemophilia : the official journal of the World Federation of Hemophilia · 2024Article
- Real World Experience with use of Coagulation Factor VIIa at an Academic Medical Center.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/HemostasisArticle
Corrections and comments
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Authors and funding
10 authors.
Funding
Abstract
introductionThe management of bleeding events (BEs) in haemophilia A (HA) and B (HB) patients with inhibitors necessitates the use of bypassing agents. The recombinant factor VIIa bypassing agent eptacog beta has demonstrated efficacy at treating BEs and managing perioperative bleeding in adults in phase three clinical studies.
aimTo provide real-world descriptions of eptacog beta use for BE treatment in patients on emicizumab or eptacog beta prophylaxis.
methodsThis is a retrospective case series of 14 patients who received eptacog beta at seven haemophilia treatment centres, with HA (n = 11) or HB (n = 3) and inhibitors or anaphylaxis to factor replacement.
resultsTwenty-four spontaneous and traumatic BEs are described (muscle hematomas, joint hemarthroses, port site, and epistaxis) involving 11 subjects. Eptacog beta was effective for acute bleed treatment as both first-line therapy and for treatment of BEs refractory to eptacog alfa in 23/24 events. When eptacog beta was used for prophylaxis, 2/3 patients reported a decreased frequency of breakthrough BEs compared with prophylactic eptacog alfa and one patient experienced a similar frequency of breakthrough BEs compared with prophylactic activated prothrombin complex concentrate. Eptacog beta provided effective bleed control for three subjects who underwent minor surgical procedures. Treatment with eptacog beta was estimated to be 46%-72% more cost-effective than eptacog alfa. No safety concerns or adverse events were reported.
conclusionsIn this case series, eptacog beta was safe, effective, and economical as first-line therapy, treatment of refractory BEs, management of perioperative bleeding, or prophylaxis in haemophilia patients with inhibitors.
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