Pharmacokinetics of a high-purity plasma-derived factor X concentrate in subjects with moderate or severe hereditary factor X deficiency

Haemophilia : the Official Journal of the World Federation of Hemophilia
S K AustinFX Investigators Group

Abstract

Hereditary factor X (FX) deficiency affects 1:500 000 to 1:1 000 000 of individuals. There are few published data on the pharmacokinetics (PK) of FX for existing treatments for FX deficiency, and no specific replacement factor concentrate exists. A high-purity plasma-derived FX concentrate (pdFX) has been developed for use as replacement therapy in subjects with hereditary FX deficiency. This analysis assessed pdFX PK after a single 25 IU kg(-1) bolus dose in subjects with hereditary moderate or severe FX deficiency (plasma FX activity [FX:C] <5 IU dL(-1) ). For a baseline PK assessment, blood samples were taken predose and at intervals up to 144 h (7 days) post dose. After ≥6 months of on-demand pdFX treatment and treatment of ≥1 bleed with pdFX, subjects underwent repeat PK assessment. Samples were assayed for plasma FX:C (measured using the clotting and chromogenic assays) and FX antigen. FX:C peaked at 0.4-0.5 h and subsequently declined over the course of 144 h with a biphasic decay curve. PK parameters observed at the baseline (n = 16) and repeat (n = 15) assessments were equivalent, therefore summary PK values were obtained by combining data from both visits (n = 31). The mean terminal half-life and incremental recovery ...Continue Reading

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Citations

Oct 21, 2016·Journal of Thrombosis and Haemostasis : JTH·F PeyvandiE Biguzzi
Jul 3, 2020·Journal of Thrombosis and Haemostasis : JTH·Karen L ZimowskiGlaivy Batsuli
Aug 15, 2021·Hematology/oncology Clinics of North America·Glaivy Batsuli, Peter Kouides
Oct 4, 2017·British Journal of Anaesthesia·E R StraussK A Tanaka

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