Hemophilia A drug appears safe, effective long-term

Efficacy

For adults and adolescents, the overall ABR was 0.66 in the individualized prophylaxis arm, 2.03 in the weekly prophylaxis arm, 1.97 in the modified prophylaxis arm, and 18.36 in the on-demand treatment arm.

For children in the individualized prophylaxis arm, the overall ABR was 0.00 in children younger than 6 and 1.56 in those ages 6 to 11. For children in the modified prophylaxis arm, the overall ABR was 6.55 in those younger than 6 and 0.00 for children 6 to 11.

A total of 566 bleeding episodes occurred in adults and adolescents who were treated prophylactically. Patients in the on-demand treatment arm experienced 262 bleeding episodes.

Overall, 90.8% of these bleeding episodes were controlled with a single infusion of rFVIIIFc, and 96.9% of the episodes were controlled with 1 or 2 infusions.

Children had a total of 51 bleeding episodes—23 among children younger than 6 and 28 among children 6 to 11.

Most of these episodes were controlled with a single infusion of rFVIIIFc—82.6% among children younger than 6 and 82.1% among children 6 to 11. And 95.7% and 89.3%, respectively, were controlled with 1 to 2 infusions.

“The results suggest prophylaxis with Eloctate shows efficacy and safety for the long-term treatment of hemophilia A,” Dr Young concluded.