Background: In patients with rare congenital fibrinogen deficiency (CFD), human fibrinogen concentrate (HFC) can be administered as prophylaxis prior to and during surgery to prevent excessive blood loss.

Aims: Data is reported on patients from two Phase 3 clinical studies using HFC to prevent surgical bleeding.

Methods: FORMA-02/FORMA-04 were international, multicenter, prospective, open-label Phase 3 studies on the efficacy and safety of HFC (Fibryga^®, Octapharma) for surgical bleeding prophylaxis in adult/adolescent and pediatric CFD patients. Hemostatic efficacy assessments were adjudicated using objective criteria by an Independent Data Monitoring Committee (IDMEAC), and overall hemostatic efficacy was assessed as success/failure for each surgery.

Results: Eleven patients (3 pediatric: <12 years; 1 adolescent: 12 years; 7 adults: ≥18 years) received HFC in 14 surgeries. Two surgeries were classified as major: 1 adult (right eye enucleation with socket reconstruction) and 1 pediatric (splenectomy). All remaining surgeries were minor. A lower mean±SD HFC dose was administered in adults prior to surgery compared to younger patients (pediatrics: 78.53±27.96 mg/kg; adolescent: 127.91 mg/kg; adults: 68.98±5.74 mg/kg). Figure 1 and Figure 2 show HFC dosing and fibrinogen activity for minor surgeries requiring single-dose treatment (target fibrinogen level = 100 mg/dL) in an adult and pediatric patient, respectively. Four minor adult surgeries required >1 HFC dose (2–5 total doses; mean±SD total dose: 132.87±29.06 mg/kg). The adult/pediatric major surgeries required 8/6 HFC infusions, respectively (loading doses: 102.56 mg/kg and 53.00 mg/kg; total doses: 225.33 mg/kg and 450.00 mg/kg). All surgeries (100%) were rated successful by the IDMEAC. One serious adverse event was considered possibly related to treatment; portal vein thrombosis in the pediatric patient undergoing splenectomy. No allergic/hypersensitivity reactions or deaths were observed.Fibrinogen activity (central lab measurements) and single HFC dose administered in a successfully treated adult patient undergoing minor surgeryFibrinogen activity (central lab measurements) and single HFC dose administered in a successfully treated pediatric patient undergoing minor surgery

Conclusions: In conclusion, HFC administration for bleeding prophylaxis during minor/major surgery was efficacious for the ultra-rare CFD in adult, adolescent and pediatric patients.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/human-fibrinogen-concentrate-for-bleeding-prophylaxis-during-surgery-in-adult-adolescent-and-pediatric-patients-with-congenital-fibrinogen-deficiency-results-from-the-forma-02-and-forma-04-clinical/