Wolfgang Miesbach Shares Real-World Evidence on Eptacog Beta from Spain
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared on LinkedIn:
”Eptacog Beta Beyond Labels: Spanish Real-World Evidence
- 8 patients | 2 Madrid centres | 3 rare bleeding disorders
- On-Label Treatments (HA + Inhibitors; n=4)
- 3/4 on concurrent emicizumab (excluded from PERSEPT trials)
- Dosing: 75 µg/kg
- Breakthrough control + thrombotic safety
- Severe iliopsoas haematoma: 70 infusions → complete resolution, zero thrombotic events
Off-Label Treatments (n=4)
- Factor VII deficiency (n=3): Perioperative surgery with 15–20 µg/kg—excellent haemostasis, zero bleeding complications
- Steroid-refractory acquired haemophilia (n=1): High-titre inhibitor case reflects poor-prognosis disease biology
No Safety Signal: Zero thrombotic/thromboembolic complications across all patients
As emicizumab, concizumab, marstacimab and fitusiran transform prophylaxis of haemophilia, eptacog beta emerges as a versatile rescue therapy.
This real-world experience expands treatment paradigms beyond current labels—pending validation from further studies.
Congratulations to the authors!”
Read the full article here.
Article: Real-World Experience with Eptacog Beta for On-Label and Off-Label Indications: The Spanish Experience
Authors: Jose Manuel Martin de Bustamante, María Isabel Rivas-Pollmar, Natalia Acedo, Nora Butta, Victor Jimenez-Yuste, Maria Teresa Álvarez-Román
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