Abstract Number: PB0698
Meeting: ISTH 2021 Congress
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.
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.
To cite this abstract in AMA style:Djambas-Khayat C, Lohade S, D'Souza F, Shamanur L, Zekavat O, Kruzhkova I, Schwartz B, Solomon C. 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 Trials [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). 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/. Accessed October 2, 2023.
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