Autor: |
Öner AF; Yüzüncü Yıl University Faculty of Medicine, Department of Pediatric Hematology, Van, Turkey., Celkan T; İstanbul University Cerrahpaşa Faculty of Medicine, Department of Pediatric Hematology and Oncology, İstanbul, Turkey., Timur Ç; İstanbul Medeniyet University, Göztepe Training and Research Hospital, Clinic of Pediatric Hematology, İstanbul, Turkey., Norton M; Bio Products Laboratory Ltd., Elstree, Hertfordshire, United Kingdom., Kavaklı K; Ege University Faculty of Medicine, Department of Pediatric Hematology, İzmir, Turkey. |
Abstrakt: |
Hereditary 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). |