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BACKGROUND: Hemophilia B is an X‐linked bleeding disorder that affects approximately 1 in 25,000 males. Therapy for acute bleeding episodes consists of transfusions of plasma‐derived (pd‐F IX) or recombinant (r‐F IX) concentrates.
STUDY DESIGN AND METHODS: A double‐blind, two‐period crossover study was initiated to assess the pharmacokinetics of pd‐F IX and r‐F IX and to address patient‐specific variables that might influence in vivo recovery. Study product was administered by a single bolus infusion (50 IU/kg) to 43 previously treated patients in the nonbleeding state, and F IX:C levels were measured over a period of 48 hours after infusion.
RESULTS: The mean in vivo recovery in the pd‐F IX group was 1.71 ± 0.73 IU per dL per IU per kg compared with 0.86 ± 0.31 IU per dL per IU per kg with r‐F IX (p ≤ 0.0001). There was a significant positive correlation (Pearsons r = 0.62, p ≤ 0.0001, 95% CI, 0.37–0.78) between the recoveries of the two products and a weak correlation between the recovery of pd‐F IX and baseline F IX:Ag levels. There was no significant difference in the terminal half‐lives of the two products.
CONCLUSIONS: The study found wide product‐ and patient‐related variability in recovery. Inherent differences among patients, including baseline F IX, may account for some of the interpatient variability. These differences should be taken into account in optimizing treatment regimens for individual patients with hemophilia B.