Abstract Number: LPB0070
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Rare Bleeding Disorders
Background: Human fibrinogen concentrate (HFC) is administered for on-demand bleeding episode (BE) treatment in patients with congenital fibrinogen deficiency (CFD): a rare disorder characterized by a lack of/low levels of functional fibrinogen.
Aims: Data is reported on the efficacy and safety of HFC for on-demand BE treatment from two Phase 3 studies in adolescent and pediatric patients (<18 years) with CFD.
Methods: FORMA-02 and FORMA-04 were international, multicenter, prospective, open-label, uncontrolled Phase 3 studies of HFC (Fibryga®, Octapharma) efficacy and safety in adult/adolescent and pediatric patients with CFD. Hemostatic efficacy was assessed by the investigators and adjudicated by an Independent Data Monitoring and Endpoint Adjudication Committee (IDMEAC) using an objective 4-point scale (excellent; good; moderate; none). All adverse events (AEs) were recorded.
Results: Six adolescent (12–17 years) and 8 pediatric (0–11 years) patients received HFC for on-demand BE treatment. HFC was administered for 11 adolescent BEs (all minor) and for 10 pediatric BEs; 8 of which were minor and 2 were major BEs (intraperitoneal bleed from spleen and knee/thigh bleed). The median (range) total dose per BE was 78.57 mg/kg (37.78–91.30) for adolescents and 73.91 mg/kg (47.45–262.50) for pediatric patients. Overall hemostatic efficacy was rated successful (excellent/good rating) for 100% of BEs by the IDMEAC.
Thirteen AEs occurred in 7 (50%) of the 14 patients receiving HFC for BE treatment. Ten AEs were mild, 2 were moderate, and 1 was severe (psoas hematoma), but was not related to treatment. No allergic/hypersensitivity reactions or deaths were observed.
Conclusions: Across two Phase 3 clinical trials, HFC was efficacious for on-demand BE treatment in adolescent and pediatric patients with CFD, which is comparable to adult patients with CFD. Hemostatic efficacy of HFC was comparable for adolescent and pediatric patients, with a favorable safety profile.
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, Peyvandi F. Efficacy and Safety of Human Fibrinogen Concentrate for Treatment of on-demand Bleeding in 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/efficacy-and-safety-of-human-fibrinogen-concentrate-for-treatment-of-on-demand-bleeding-in-adolescent-and-pediatric-patients-with-congenital-fibrinogen-deficiency-results-from-the-forma-02-and-forma/. Accessed December 11, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/efficacy-and-safety-of-human-fibrinogen-concentrate-for-treatment-of-on-demand-bleeding-in-adolescent-and-pediatric-patients-with-congenital-fibrinogen-deficiency-results-from-the-forma-02-and-forma/