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

Abstract

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).

“I have been refused to be treated by three dentists”: Barriers to patient care

“…The inherited type is an autosomal recessive type, which mainly occurs due to nonsense mutations in the F10 gene, 4,6 which is why it is sometimes known as F10 deficiency. It is mostly seen in nations with a high percentage of consanguineous marriage 7 . Affected persons with the homozygous deficiency form show severe bleeding symptoms at a younger age, while persons with the heterozygous form develop symptoms of hemorrhage in the case of severe trauma or if they undergo a major surgical procedure.…”

“…The most common manifestations of factor X deficiency comprise bleeding of the joints, hemorrhage of muscles or mucous membranes, prolonged nasal bleeding, easy bruising, menorrhagia, and spontaneous internal or external haemorrhage 2,7,9 . Patients with IBD reported difficulty in accessing primary dental care, either due to their medical diseases or their related barriers 10 .…”