Abstract Number: PB1176
Meeting: ISTH 2020 Congress
Background: Congenital fibrinogen deficiency (CFD) is a rare, inherited bleeding disorder affecting hemostasis. Patients experience severe and/or frequent bleeding episodes (BEs). Treatment with human fibrinogen concentrate (HFC) can prevent and arrest bleeding. The rarity of CFD makes it difficult to perform prospective pediatric clinical trials.
Aims: FORMA-04, was a multinational, multicenter, prospective, open-label, uncontrolled, Phase 3 study to determine the efficacy, safety and pharmacokinetics (PK) of HFC (Fibryga®, Octapharma AG) for treatment of acute BEs and surgical prophylaxis in pediatric patients with CFD.
Methods: Hemostatic efficacy was assessed based on an objective 4-point scale with final adjudication by an Independent Adjudication Committee (IDMEAC). PK blood samples were collected at baseline and at 1- and 3-hours on day 2, 5, 7, 10 and 14 post-infusion. Safety was assessed by monitoring adverse events (AEs).
Results: Fourteen afibrinogenemic patients (mean age of 6.0 years, range 1.0-10.0 years) were enrolled. Eight patients received HFC for the treatment of 10 BEs (8 minor, 2 major). Overall hemostatic efficacy for on-demand treatment of BEs was rated as 100% successful (Figure). Haemostatic efficacy of three surgeries (2 minor, 1 major) in 3 patients was rated as 100% successful. Mean (±SD) PK parameters, as determined by measuring fibrinogen activity are shown in the Table. Overall, 10 AEs occurred in 4 (28.6%) patients, 2 of which (pyrexia and portal vein thrombosis) were in the same patient and assessed as possibly related to treatment. The latter AE was classified as serious, occurring after splenectomy for a spontaneous spleen rupture, leading to discontinuation from the study. No allergic/hypersensitivity reactions and no deaths were observed.
Conclusions: This data set demonstrates that HFC treatment was efficacious and safe for the on-demand treatment of acute BEs and surgical prophylaxis in pediatric patients with congenital afibrinogenemia. PK profile was comparable to that in adults.
|Incremental IVR, mg/dL/(mg/kg)||1.592||0.3224||1.599||1.155-2.259|
[Table. PK Parameters for HFC Determined by Fibrinogen Antigen (N=13)]
To cite this abstract in AMA style:Lohade SD, D'Souza F, Latha GS, Khayat C, Zekavat O, Schwartz BA, Kruzhkova I, Solomon C, Knaub S, Peyvandi F. Efficacy and Safety of Fibrinogen Concentrate for On-Demand Treatment of Acute Bleeding and Surgical Prophylaxis in Pediatric Patients with Congenital Fibrinogen Deficiency: Results from the FORMA-04 Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/efficacy-and-safety-of-fibrinogen-concentrate-for-on-demand-treatment-of-acute-bleeding-and-surgical-prophylaxis-in-pediatric-patients-with-congenital-fibrinogen-deficiency-results-from-the-forma-04/. Accessed September 21, 2023.
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