Efficacy, safety and pharmacokinetics of a new high‐purity factor X concentrate in subjects with hereditary factor X deficiency

Abstract

These results demonstrate that a dose of 25 IU kg(-1) pdFX is safe and efficacious for on-demand treatment and short-term prophylaxis in subjects with moderate or severe hereditary FX deficiency.

Prophylactic treatment of bleeding episodes in children <12 years with moderate to severe hereditary factor X deficiency (FXD): Efficacy and safety of a high‐purity plasma‐derived factor X (pdFX) concentrate

“…Of the 3 bleeds that were assessed for efficacy, pdFX treatment efficacy was rated as excellent. These results are consistent with a previous study conducted in subjects aged ≥12 years in which pdFX successfully treated bleeds in 97% of subjects, and 83% of bleeds were treated with a single infusion …”

“…During extended prophylactic use of pdFX in subjects who had previously received treatment with other clotting factors—including 3 subjects who had received pdFX for compassionate use prior to study entry—no evidence was seen in this study population for the development of FX inhibitors. These data are consistent with previous pdFX studies in patients ≥12 years old with moderate to severe FXD, who also showed no sign of inhibitor development . In addition, no cases of FX inhibitor development have been reported in postmarketing safety surveillance.…”

“…Several studies have been published on the use of pdFX in subjects ≥12 years old with hereditary FXD . However, data describing the use of FX‐specific treatments in children <12 years old are scarce.…”

Pharmacokinetics of a high‐purity plasma‐derived factor X concentrate in subjects with moderate or severe hereditary factor X deficiency

“…In summary, the PK characteristics of pdFX presented here are consistent with other coagulation factors used as replacement therapy in inherited bleeding disorders and are consistent with the observed haemostatic efficacy of the product in subjects aged ≥12 years with hereditary FX deficiency [14]. In addition, the 30-h t ½ of pdFX may facilitate a once-or twice-weekly prophylactic dosing schedule, depending on the required trough FX:C level.…”

Congenital Factor X deficiency in women: A systematic review of the literature

“…FX levels should be monitored to ensure therapeutic levels of 0.4 IU/mL have been achieved at the time of delivery (level C recommendation). PCC 20‐40 IU/kg (or factor X concentrate if available) to achieve factor X activity more than 0.4 IU/mL, further doses of PCC 10‐20 IU/kg once daily to maintain factor X activity more than 0.3 IU/mL for at least 3 days . Tranexamic acid 1 g 6 hourly is also recommended till the lochial loss is very light.…”