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

Ahmet F Öner, Tiraje Celkan, Çetin Timur, Miranda Norton, Kaan Kavaklı, Ahmet F Öner, Tiraje Celkan, Çetin Timur, Miranda Norton, Kaan Kavaklı

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).

Keywords: Clinical trial; Clotting factor concentrate; Efficacy; Factor X deficiency; Orphan drug Safety..

Conflict of interest statement

Conflict of Interest: M.N. is an employee of Bio Products Laboratory Ltd. K.K. has received investigational support from Bio Products Laboratory Ltd. Other authors of this paper have no conflicts of interest, including specific financial interests, relationships, and/or affiliations relevant to the subject matter or materials included.

Figures

Figure 1. Summary of bleeding episodes treated…
Figure 1. Summary of bleeding episodes treated with plasma-derived FX (Turkish cohort). pdFX: plasma-derived FX.

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