Austin, S.K.Kavakli, K.Norton, M.Peyvandi, F.Shapiro, A.Álvarez Román, M.-T.Timur, C.2025-05-102025-05-1020161351-821610.1111/hae.128932-s2.0-84962028764https://doi.org/10.1111/hae.12893https://hdl.handle.net/20.500.14720/4950Introduction: Hereditary factor X (FX) deficiency is a rare bleeding disorder affecting 1:500 000 to 1:1 000 000 of individuals. Until recently, no specific replacement factor concentrate was available. Aim: The aim of this study was to assess safety and efficacy of a new, high-purity plasma-derived FX concentrate (pdFX) in subjects with hereditary FX deficiency. Methods: Subjects aged ≥12 years with moderate or severe FX deficiency (plasma FX activity <5 IU dL-1) received 25 IU kg-1 pdFX as on-demand treatment or short-term prophylaxis for 6 months to 2 years. Subjects assessed pdFX efficacy for each bleed; at end-of-study, investigators assessed overall pdFX efficacy. Blood samples for pharmacokinetic analysis were obtained at baseline and ≥6 months. Safety was assessed by adverse events (AEs), inhibitor development and changes in laboratory parameters. Results: Sixteen enrolled subjects (six aged 12-17 years; 10 aged 18-58 years) received a total of 468 pdFX infusions. In the 187 analysed bleeds, pdFX efficacy was categorized as excellent, good, poor or unassessable in 90.9%, 7.5%, 1.1% and 0.5% of bleeds respectively; 83% of bleeds were treated with one infusion. For pdFX, mean (median; interquartile range) incremental recovery and half-life were 2.00 (2.12; 1.79-2.37) IU dL-1 per IU kg-1 and 29.4 (28.6; 25.8-33.1) h respectively. No serious AEs possibly related to pdFX or evidence of FX inhibitors were observed, and no hypersensitivity reactions or clinically significant trends were detected in laboratory parameters. Conclusion: These results demonstrate that a dose of 25 IU kg-1 pdFX is safe and efficacious for on-demand treatment and short-term prophylaxis in subjects with moderate or severe hereditary FX deficiency. © 2016 John Wiley & Sons Ltd.eninfo:eu-repo/semantics/openAccessClinical TrialClotting Factor ConcentrateEfficacyFactor X DeficiencyOrphan DrugSafetyArticle223Q2Q241942527197801