Real-World Experience with a Human Fibrinogen Concentrate: Clinical Data from Adult and Pediatric Patients Requiring Fibrinogen for Bleeding Control and Prevention

Authors: Stéphan, F; Gutermann, L; Bourget, S; Djabarouti, S; Berdugo, J; Fardini, Y; Clerson, P; Hébert, G; Belmokhtar, C

Affiliations: Hôpital Marie Lannelongue, Groupe Hospitalier Paris Saint Joseph, Service de réanimation adultes, Le Plessis Robinson, France. Centre Hospitalier de Valence, Service Pharmacie, Valence, France. Groupe Hospitalier Sud, CHU de Bordeaux, Service Pharmacie, Pessac, France. Hôpital Saint-Joseph, Service Pharmacie, Marseille, France. Soladis Clinical Studies, Roubaix, France. Octapharma France SAS, Boulogne-Billancourt, France.

Publication: Journal of clinical pharmacology; 2023

Abstract: Human fibrinogen concentrate (Fibryga®, Octapharma AG) received temporary approval for fibrinogen replacement therapy in France (2017), subsequently obtaining full approval for congenital and acquired hypofibrinogenemia. We evaluated real-world use for on-demand treatment of bleeding and prophylaxis to enhance our knowledge on fibrinogen concentrate as an option for fibrinogen replacement. Data was retrospectively collected from adult and pediatric patients with fibrinogen deficiency. Primary endpoint was indication for fibrinogen concentrate use; the secondary endpoint was treatment success for on-demand treatment/perioperative prophylaxis. The study included 150 adult (median [range] age 62 years [18-94]) and 50 pediatric patients (3 years [0.01-17]) with acquired fibrinogen deficiency. Fibrinogen concentrate was administered to 47.3% adults for non-surgical bleeding, 22.7% for surgical bleeding, and 30.0% for perioperative prophylaxis, with 4.0% for surgical bleeding and 96.0% for perioperative prophylaxis in pediatric patients. Cardiac surgeries accounted for 79.5/75.0% perioperative prophylaxis and 82.4/100.0% surgical bleeding cases in adult/pediatric patients, respectively. Mean±SD (median) total fibrinogen doses were 3.06±1.69 g (32.61 mg/kg), 2.09±1.36 g (22.99 mg/kg), and 2.36±1.25 g (29.67 mg/kg) for adult non-surgical bleeding, surgical bleeding, and perioperative prophylaxis; with 0.75±0.35 g (47.64 mg/kg) and 0.83±0.62 g (55.56 mg/kg) for pediatric surgical bleeding and perioperative prophylaxis, respectively. Treatment success in adults was 85.7/97.1/93.3%, and 50.0/87.5% in pediatrics for non-surgical bleeding (adults only), surgical bleeding, and perioperative prophylaxis, respectively. Fibrinogen concentrate demonstrated favorable effectiveness and safety across age groups. This study contributes to evidence supporting fibrinogen concentrate for bleeding control/prevention in real-world clinical practice, particularly for patients with acquired fibrinogen deficiency.