Abstract | INTRODUCTION: Hereditary factor X (FX) deficiency is a rare bleeding disorder affecting 1:500 000 to 1:1 000 000 of individuals. Until recently, no specific replacement factor concentrate was available. AIM: The aim of this study was to assess safety and efficacy of a new, high-purity plasma-derived FX concentrate (pdFX) in subjects with hereditary FX deficiency. METHODS: Subjects aged ≥12 years with moderate or severe FX deficiency (plasma FX activity <5 IU dL(-1) ) received 25 IU kg(-1) pdFX as on-demand treatment or short-term prophylaxis for 6 months to 2 years. Subjects assessed pdFX efficacy for each bleed; at end-of-study, investigators assessed overall pdFX efficacy. Blood samples for pharmacokinetic analysis were obtained at baseline and ≥6 months. Safety was assessed by adverse events (AEs), inhibitor development and changes in laboratory parameters. RESULTS: Sixteen enrolled subjects (six aged 12-17 years; 10 aged 18-58 years) received a total of 468 pdFX infusions. In the 187 analysed bleeds, pdFX efficacy was categorized as excellent, good, poor or unassessable in 90.9%, 7.5%, 1.1% and 0.5% of bleeds respectively; 83% of bleeds were treated with one infusion. For pdFX, mean (median; interquartile range) incremental recovery and half-life were 2.00 (2.12; 1.79-2.37) IU dL(-1) per IU kg(-1) and 29.4 (28.6; 25.8-33.1) h respectively. No serious AEs possibly related to pdFX or evidence of FX inhibitors were observed, and no hypersensitivity reactions or clinically significant trends were detected in laboratory parameters. CONCLUSION: 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.
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Authors | S K Austin, K Kavakli, M Norton, F Peyvandi, A Shapiro, FX Investigators Group |
Journal | Haemophilia : the official journal of the World Federation of Hemophilia
(Haemophilia)
Vol. 22
Issue 3
Pg. 419-25
(May 2016)
ISSN: 1365-2516 [Electronic] England |
PMID | 27197801
(Publication Type: Clinical Trial, Phase III, Journal Article, Multicenter Study)
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Copyright | © 2016 John Wiley & Sons Ltd. |
Chemical References |
- Antibodies, Neutralizing
- Factor X
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Topics |
- Adolescent
- Adult
- Antibodies, Neutralizing
(blood)
- Blood Coagulation Tests
- Child
- Factor X
(adverse effects, pharmacokinetics, therapeutic use)
- Factor X Deficiency
(congenital, drug therapy, pathology)
- Female
- Half-Life
- Hemorrhage
(prevention & control)
- Humans
- Male
- Menorrhagia
(prevention & control)
- Middle Aged
- Severity of Illness Index
- Treatment Outcome
- Young Adult
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