ArticleHaemophilia : the official journal of the World Federation of Hemophilia2021
Efficacy and safety of fibrinogen concentrate for on-demand treatment of bleeding and surgical prophylaxis in paediatric patients with congenital fibrinogen deficiency.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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Who cites it
7 citing papers in PubMed, 19 citations in OpenAlex.
- Trial
- Pharmacokinetics, Hemostatic Efficacy, and Safety of a New Human Fibrinogen Concentrate in Adult and Pediatric Patients with Congenital Fibrinogen Deficiency.Thrombosis and haemostasis · 2026Article
- The thrombotic paradox in congenital fibrinogen deficiencies: from pathophysiology to practice.Research and practice in thrombosis and haemostasis · 2025Review
- Treatment of Congenital Afibrinogenemia in a Neonate With Critical Pulmonary Stenosis.The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG · 2023Article
- Differences in coagulation-relevant parameters: Comparing cryoprecipitate and a human fibrinogen concentrate.PloS one · 2023Article
- Analysis of fibrinogen concentrate pharmacokinetics and dosing for bleeds and surgery in adults, adolescents, and children with congenital afibrinogenaemia and hypofibrinogenaemia.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Article
- Disorders of Fibrinogen and Fibrinolysis.Hematology/oncology clinics of North America · 2021Review
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Authors and funding
10 authors at 8 institutions in 6 countries.
Funding
Abstract
backgroundCongenital fibrinogen deficiency (CFD) is a rare, inherited disorder affecting normal blood clotting function, where patients can experience severe and/or frequent bleeding episodes (BEs). Treatment with human fibrinogen concentrate (HFC) can prevent/arrest bleeding. There is a need for more data on the efficacy, pharmacokinetics (PK) and safety of HFC treatment in paediatric patients with CFD.
methodsHaemostatic efficacy of HFC (Fibryga
resultsOf 14 patients receiving HFC (median [range] age 6.0 years [1.0-10.0]), eight received HFC for 10 BEs, three for surgical prophylaxis and 13 for PK. The IDMEAC rated haemostatic efficacy as 100% successful for on-demand BE treatment (95% CI 69.15-100.00) and surgical prophylaxis (95% CI 29.24-100.00). After a mean first dose of 70.78 mg/kg for BEs, mean (±SD) MCF significantly increased from pre-treatment to 1-hour post-infusion (3.3 mm [±1.77]; P = 0.0002), coinciding with haemostatic efficacy. PK parameters were favourable. Two possibly related adverse events occurred, including one serious (portal vein thrombosis). No allergic/hypersensitivity reactions or deaths were observed.
conclusionHFC treatment for on-demand treatment of BEs and surgical prophylaxis was efficacious for this ultra-rare paediatric population with congenital afibrinogenaemia and showed a favourable PK and safety profile.
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