Plasma-derived human factor X concentrate for the treatment of patients with hereditary factor X deficiency.
Haemophilia
; 30(1): 59-67, 2024 Jan.
Article
de En
| MEDLINE
| ID: mdl-37902395
ABSTRACT
INTRODUCTION:
Hereditary factor X (FX) deficiency (HFXD) is an autosomal recessive rare bleeding disorder that leads to defects in the FX protein. Depending on the degree of deficiency, patients may be at risk of life-threatening bleeding episodes. Historical treatments for FX deficiency include prothrombin complex concentrates, which can increase the risk of thrombosis, and fresh frozen plasma, which can cause volume overload and transfusion reactions. Plasma-derived FX (pdFX), a single-factor, high-purity, high-potency human FX treatment, was approved in 2015 in the United States and in 2016 in Europe for on-demand treatment and prophylaxis of bleeding episodes and perioperative management of patients with HFXD.METHODS:
Five studies that examined the use of pdFX in patients with mild (plasma FX activity [FXC] ≥5 IU/dL), moderate (FXC ≥1 and <5 IU/dL), or severe (FXC < 1 IU/dL) HFXD were reviewed TEN01, TEN02 and TEN03 were prospective, open-label, multicentre, nonrandomised studies, and TEN05 and TEN06 were multicentre retrospective studies.RESULTS:
When used as an on-demand treatment, pdFX was judged by investigators to be successful in treating 41/42 (97.6%), 2/3 (66.6%) and 79/79 (100%) bleeds in TEN01, TEN02 and TEN05, respectively. When used prophylactically, pdFX was judged 'excellent' for the prevention of bleeds in nine (100%) and eight (100%) patients in TEN02 and TEN05, respectively. Perioperative treatment and pharmacokinetics were also assessed. pdFX was safe and well tolerated.CONCLUSIONS:
Together, these studies support the use of pdFX for on-demand treatment of bleeding, routine prophylaxis, and perioperative management of bleeding in patients with HFXD.Mots clés
Texte intégral:
1
Collection:
01-internacional
Base de données:
MEDLINE
Sujet principal:
Facteur X
/
Déficit en facteur X
Limites:
Humans
Langue:
En
Journal:
Haemophilia
Sujet du journal:
HEMATOLOGIA
Année:
2024
Type de document:
Article
Pays d'affiliation:
États-Unis d'Amérique