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]
Küçük Resim Yok
Tarih
2018
Yazarlar
Dergi Başlığı
Dergi ISSN
Cilt Başlığı
Yayıncı
Turkish Society of Hematology
Erişim Hakkı
info:eu-repo/semantics/openAccess
Özet
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). ©2018 by Turkish Society of Hematology
Açıklama
Anahtar Kelimeler
Clinical trial, Clotting factor concentrate, Efficacy, Factor X deficiency, Orphan drug, Safety
Kaynak
Turkish Journal of Hematology
WoS Q Değeri
Scopus Q Değeri
Q3
Cilt
35
Sayı
2
