title: The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B.
abstract: This international clinical trial evaluated the safety and efficacy of recombinant factor IX (rFIX) in previously untreated patients (PUPs) with severe or moderately severe hemophilia B (FIX activity, < or = 3 IU/dL). Sixty-three PUPs aged younger than 1 month to 14 years received rFIX (median treatment duration, 37 months; range, 4-64 months). Mean rFIX recovery (0.68 +/- 0.27 IU/dL per IU/kg) remained constant over 5 years and was similar in infants (1 month to < 2 years) and children (2 to < 12 years). Fifty-four PUPs used rFIX (median dose, 62.7 IU/kg per infusion; range, 8.2-292 IU/kg) to treat 997 hemorrhages. Bleeding was well controlled, with 75% of hemorrhages requiring only one rFIX infusion. Response to rFIX was "excellent" or "good" in 94% of cases. Effective hemostasis was achieved in 32 PUPs receiving rFIX for routine prophylaxis, with 91% of prophylaxis responses rated "excellent." rFIX administered for 30 surgical procedures in 23 PUPs achieved hemostasis for all rated procedures. Five patients experienced allergic-type manifestations, including 2 (3%) patients who developed FIX inhibitors (both > 5 BU/dL). rFIX was well tolerated, with no associated thrombotic events or evidence of viral transmission. These data indicate that rFIX is a safe and effective treatment for PUPs with hemophilia B.
authors.affiliation: Indiana Hemophilia and Thrombosis Center, Indianapolis 46260, USA. ashapiro@ihtc.org
authors.forename: Amy D
authors.initials: AD
authors.lastname: Shapiro
authors.forename: Jorge
authors.initials: J
authors.lastname: Di Paola
authors.forename: Alice
authors.initials: A
authors.lastname: Cohen
authors.forename: K John
authors.initials: KJ
authors.lastname: Pasi
authors.forename: Margaret A
authors.initials: MA
authors.lastname: Heisel
authors.forename: Victor S
authors.initials: VS
authors.lastname: Blanchette
authors.forename: Thomas C
authors.initials: TC
authors.lastname: Abshire
authors.forename: W Keith
authors.initials: WK
authors.lastname: Hoots
authors.forename: Jeanne M
authors.initials: JM
authors.lastname: Lusher
authors.forename: Claude
authors.initials: C
authors.lastname: Negrier
authors.forename: Chantal
authors.initials: C
authors.lastname: Rothschild
authors.forename: David A
authors.initials: DA
authors.lastname: Roth
mesh_terms: D000293:Adolescent; D016063:Blood Loss, Surgical / Q000517:prevention & control; D002648:Child; D002675:Child, Preschool; D005164:Factor IX / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D015658:HIV Infections / Q000175:diagnosis; D002836:Hemophilia B / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control*; D006506:Hepatitis A / Q000175:diagnosis; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000009:adverse effects; D012720:Severity of Illness Index; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D005164:Factor IX