Use of a High-Purity Factor X Concentrate in Turkish Subjects with Hereditary Factor X Deficiency: Post Hoc Cohort Subanalysis of a Phase 3 Study

WOS: 000433166100006PubMed ID: 29545231Hereditary 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 cohortBio Products Laboratory (Elstree, UK)Bio Products Laboratory (Elstree, UK) provided support for this study and funding for medical writing and editorial support in the development of this manuscript. A.F.O.: Received educational support from Pfizer. M.N.: Employee of Bio Products Laboratory. K.K.: Advisory board member for Bayer, Novo Nordisk, Pfizer, and Shire; received educational and investigational support from Bayer, Bio Products Laboratory, CSL Behring, Novo Nordisk, Octapharma, Pfizer, and Shire