Oner, Ahmet F.Celkan, TirajeTimur, CetinNorton, MirandaKavakli, Kaan2025-05-102025-05-1020181300-77771308-526310.4274/tjh.2017.04462-s2.0-85047784869https://doi.org/10.4274/tjh.2017.0446https://hdl.handle.net/20.500.14720/7600Celkan, Tulin Tiraje/0000-0001-7287-1276Hereditary factor X (FX) deficiency is a rare bleeding disorder more prevalent in countries with high rates of consanguineous marriage. In a prospective, open-label, multicenter phase 3 study, 25 IU/kg plasma-derived factor X (pdFX) was administered as on-demand treatment or short-term prophylaxis for 6 months to 2 years. In Turkish subjects (n=6), 60.7% of bleeds were minor. A mean of 1.03 infusions were used to treat each bleed, and mean total dose per bleed was 25.38 IU/kg. Turkish subjects rated pdFX efficacy as excellent or good for all 84 assessable bleeds; investigators judged overall pdFX efficacy to be excellent or good for all subjects. Turkish subjects had 51 adverse events; 96% with known severity were mild/moderate, and 1 (infusion-site pain) was possibly pdFX-related. These results demonstrate that 25 IU/kg pdFX is safe and effective in this Turkish cohorteninfo:eu-repo/semantics/openAccessClinical TrialClotting Factor ConcentrateEfficacyFactor X DeficiencyOrphan DrugSafetyArticle352Q3Q312913329545231WOS:000433166100006