The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B.
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.
Shapiro
Amy D
AD
Indiana Hemophilia and Thrombosis Center, Indianapolis 46260, USA. ashapiro@ihtc.org
Di Paola
Jorge
J
Cohen
Alice
A
Pasi
K John
KJ
Heisel
Margaret A
MA
Blanchette
Victor S
VS
Abshire
Thomas C
TC
Hoots
W Keith
WK
Lusher
Jeanne M
JM
Negrier
Claude
C
Rothschild
Chantal
C
Roth
David A
DA
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
D011994:Recombinant Proteins; D005164:Factor IX