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 [Kalıtsal faktör x eksikliği olan türk hastalarda yüksek saflıkta faktör x konsantresi kullanımı: Faz 3 çalışmasının post hoc kohort alt analizi]

Öner A.F.Celkan T.Timur Ç.Norton M.Kavaklı K.2019-10-272019-10-2720181300-7777https://doi.org/10.4274/tjh.2017.0446https://hdl.handle.net/11454/25312Hereditary 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 cohort (ClinicalTrials.gov identifier: NCT00930176). ©2018 by Turkish Society of Hematologyen10.4274/tjh.2017.0446info:eu-repo/semantics/openAccessClinical trialClotting factor concentrateEfficacyFactor X deficiencyOrphan drugSafetyUse of a high-purity factor x concentrate in turkish subjects with hereditary factor x deficiency: Post hoc cohort subanalysis of a phase 3 study [Kalıtsal faktör x eksikliği olan türk hastalarda yüksek saflıkta faktör x konsantresi kullanımı: Faz 3 çalışmasının post hoc kohort alt analizi]Article35212913329545231Q3