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Blood, 15 December 2001, Vol. 98, No. 13, pp. 3600-3606
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Human recombinant factor IX: safety and efficacy studies in
hemophilia B patients previously treated with plasma-derived factor IX
concentrates
David A. Roth,
Craig M. Kessler,
K. John Pasi,
Bonita Rup,
Suzanne G. Courter, and
Karen L. Tubridy for the Recombinant Factor IX Study Group
From the Beth Israel Deaconess Medical Center, Harvard
Medical School, Boston, MA; Georgetown University Medical Center,
Georgetown University, Washington, DC; Royal Free Hospital, Royal Free
Hampstead NHS Trust, London, United Kingdom; and Genetics Institute,
Cambridge, MA. A complete list of the members of the Recombinant Factor
IX Study Group is provided in the appendix.
Human plasma-derived factor IX (pdFIX) concentrates are routinely
used to treat patients with hemophilia B, an X-linked bleeding disorder
that affects 1 in 30 000 males, but concerns remain regarding transmission of blood-borne pathogens. Therefore, the safety and efficacy of recombinant human factor IX (rFIX) were evaluated. A
20-center international trial was conducted in previously treated patients with severe or moderate (< 5 IU/dL factor IX activity) hemophilia B. Participants received rFIX for pharmacokinetic studies, treatment of or prophylaxis against hemorrhage, or surgical hemostasis, and were assessed at 3-month intervals for 2 years. Fifty-six subjects
were treated. Mean incremental rFIX recovery was 0.75 IU/dL per IU/kg,
30% lower than expected for pdFIX, although the mean half-life was
similar. Pharmacokinetic parameters were stable over time. Somewhat
lower recoveries were seen in subjects younger than 15 years of age and
in those with no detectable factor IX antigen. A total of 7362 infusions of rFIX were administered. All 1796 hemorrhages were
controlled, 80.9% of which required only one rFIX infusion. Effective
hemostasis was also achieved in prophylactic and surgical settings. One
individual developed a low titer (1.2 Bethesda unit) transient
inhibitor that spontaneously resolved. rFIX was not associated with
serious adverse events, thrombogenicity, or virus transmission. rFIX is
safe and effective for the treatment of hemophilia B. Despite a lower
recovery compared with pdFIX, rFIX controlled hemorrhage in a wide
variety of settings and may provide a safety advantage in terms of risk
from blood-borne pathogens.
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Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates
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Blood 2002 100: 4242.
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