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PLASTIC RECONSTRUCTIVE AND REGENERATIVE SURGERY

Abstract

People with severe hemophilia B (PWHB) require intravenous anti-hemorrhagic prophylaxis with coagulation factor IX (FIX) to prevent acute bleeding and chronic arthropathy. Inhibitory antibodies (INH) against FIX develop in 3–5% of such patients, making their clinical management hard. In these scenarios bypassing agents are crucial for managing acute bleeding episodes. Eptacog beta is a recombinant activated factor VII with a distinct post-translational modification profile. In Europe it is indicated for the treatment of bleeds in patients aged ≥12 years with congenital hemophilia A or B and high-titer inhibitors, or low-titer inhibitors expected to show a high anamnestic response or refractoriness to increased factor dosing. We describe the first use of eptacog beta to treat bleeding in a PWHB with INH receiving prophylaxis with an anti-tissue factor pathway inhibitor (TFPI). We report the case of an 11-year-old male with severe hemophilia B (FIX <1%) with a history of high-titer INH and anaphylactoid reaction. INH appeared after five doses of FIX given as primary prophylaxis starting at 18 months old. In October 2025, secondary prophylaxis with concizumab (a subcutaneous anti-TFPI antibody) was initiated. Three months later, to control right elbow hemarthrosis the boy was administered intravenous eptacog-beta (Cevenfacta®), in off-label use due to his age, at a dosage of 75 μg/kg. This induced a hemostatic response, leading to full resolution of the pain and functional impairment. The infusion was well tolerated by the patient. This is the first literature report of eptacog beta use in a child <12 years receiving concizumab prophylaxis. With all the caveats of generalizing information from a single case, the efficacy, favorable safety profile and good tolerability observed in our child suggest that eptacog beta could be investigated further as a valid therapeutic option for managing breakthrough bleeding episodes in clinically complex patients.

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Authors

Giuseppe Lassandro Interdisciplinary Medicine Department, University of Bari “Aldo Moro”, Pediatric Unit “B.Trambusti” AOUC Policlinico-Giovanni XXIII, Bari, Italy ORCID

Luisa Laterza Interdisciplinary Medicine Department, University of Bari “Aldo Moro”, Pediatric Unit “B.Trambusti” AOUC Policlinico-Giovanni XXIII, Bari, Italy

Cornelia Guerrino BIOVIIIx, Napoli, Italy ORCID

Valentina Palladino Interdisciplinary Medicine Department, University of Bari “Aldo Moro”, Pediatric Unit “B.Trambusti” AOUC Policlinico-Giovanni XXIII, Bari, Italy

Paola Giordano Interdisciplinary Medicine Department, University of Bari “Aldo Moro”, Pediatric Unit “B.Trambusti” AOUC Policlinico-Giovanni XXIII, Bari, Italy

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