Experience of a new high‐purity factor X concentrate in subjects with hereditary factor X deficiency undergoing surgery

Abstract

These data demonstrate that pdFX is safe and effective as replacement therapy in five subjects with mild-to-severe FX deficiency undergoing surgery on seven occasions.

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

“…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.…”

“…Several studies have been published on the use of pdFX in subjects ≥12 years old with hereditary FXD. [11][12][13]16 However, data describing the use of FX-specific treatments in children <12 years old are scarce. Prior to the start of the current trial, 3 subjects had received pdFX as routine prophylaxis for 1-4 years on a compassionate use basis, but pharmacokinetic and efficacy data were not collected prospectively.…”

“…All reported AEs were mild or moderate in severity, and the 2 serious FXD, who also showed no sign of inhibitor development. 12,13 In ad- 20,21,23 No known reports exist on the development of alloantibodies following replacement therapy for hereditary FXD, and a previous study of prophylactic treatment for up to 2 years reported no development of inhibitors. 12 However, it remains possible that long-term exposure to pdFX may lead to inhibitor development in some patients, and all individuals with FXD who receive pdFX on a prophylactic basis should be monitored for inhibitors.…”

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

“…A study of 16 subjects with moderate or severe FX deficiency (plasma FX:C <5 IU dL −1 ) demonstrated the safety and efficacy of pdFX at doses of 25 IU kg −1 administered to treat spontaneous, menorrhagic and traumatic bleeding . Similar results were observed when pdFX was administered to five subjects with mild‐to‐severe FX deficiency (plasma FX:C <20 IU dL −1 ) for haemostatic cover before and after surgery . Independently, breakthrough bleeding in a 9‐year‐old child was shown to be effectively prevented with pdFX maintained at a trough level of 6 IU dL −1 (72 h post dose) over a 3‐year period , supporting the observation that low plasma FX levels are adequate for haemostasis in some subjects.…”