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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

1Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy, 2Hotel Dieu de France Hospital, Beirut, Lebanon, 3Sahyadri Specialty Hospital, Pune, India, 4St. John’s Medical College Hospital, Bangalore, India, 5S.S Institute of Medical Science and Research Center, Davangere, India, 6Hematology Research Center, Nemazee Hospital, Shiraz University of Medical Sciences, Shiraz, Iran, Islamic Republic of, 7Octapharma AG, Lachen, Switzerland, 8Octapharma, Hoboken, United States, 9Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan, Italy

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.

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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/