Efficacy and Safety
Bleed control for when you need it—
Proven bleed control
of bleeds were successfully controlled with one or two infusions in a clinical study
Among 53 adults and adolescents who were treated on demand for 187 bleeding episodes:
- 70.6% of responses were rated excellent or good
- 24.1% of responses were rated moderate
- 2.7% were rated no response
- 2.7% were not rated
Excellent: abrupt pain relief and/or improvement in signs of bleeding within approximately 8 h after a single infusion; Good: definite pain relief and/or improvement in signs of bleeding within approximately 8 h after an infusion, but possibly requiring more than one infusion for complete resolution; Moderate: probable or slight beneficial effect within approximately 8 h after the first infusion, usually requiring more than one infusion; or No Response: no improvement, or condition worsens. (Response rating for 5 hemorrhages not reported.)
Study design: Results based on 94 PTPs median age of 24 years, range 12-60 years of age, with ≥150 previous exposure days with baseline FVIII activity level of ≤2%; XYNTHA was administered at a dose of 30 ± 5 IU/kg 3 times a week with provisions for dose escalation based on prespecified criteria (7 dose escalations were prescribed).
ACTIVE IS going to my daughter's field hockey game, putting some work in on the bike, and running soccer drills with my son this weekend.
Demonstrated bleed control during and after surgery
of bleed control ratings were "excellent" or "good" during and up to 1 hour after surgery
In the open-label clinical study of 30 adults and adolescents:
- 72% of responses were rated excellent
- 28% of responses were rated good
Surgical prophylaxis study: Open-label study (n=30) for surgical prophylaxis in PTPs with severe or moderately severe hemophilia A (FVIII:C ≤2%) undergoing major surgical procedures. Results reported here for 25 patients who received at least one dose of XYNTHA replacement therapy over at least 6 days post-surgery.
Efficacy responses were assessed as follows: Excellent: Achieved hemostasis comparable to that expected after similar surgery in a patient without hemophilia. Good: Prolonged time to hemostasis, with somewhat increased bleeding compared with that expected after similar surgery in a patient without hemophilia. End of initial postoperative period was date of discharge or postoperative Day 6, whichever occurred later.
ACTIVE IS a soccer practice on Saturday, kickball with my friends at recess, and the movies with the family next weekend.
Proven bleed control in adolescents and children
Bleed control in adolescents (12 to <16 years)
of bleeds were controlled with one or two on‑demand infusions in a clinical study
In a clinical study, 66 bleeding episodes were treated on demand with XYNTHA:
- 57.6% of responses were rated excellent or good
- 36.4% of responses were rated moderate
- 6.1% were not rated
Study design: The open-label study of PTPs with severe or moderately severe hemophilia A (FVIII:C ≤2%; ≥150 previous exposure days) included 17 adolescents 12 to <16 years of age who received XYNTHA for on-demand and follow-up treatment. Results reported here for 10 patients (66 bleeding episodes). The median dose/infusion was 47 IU/kg.
Bleed control in children (<6 years)
of bleeds were controlled with one or two on‑demand infusions of XYNTHA
In a clinical study, 493 bleeding episodes were treated on demand with XYNTHA:
- 94.9% of responses were rated excellent or good
- 4.5% of responses were rated moderate
- 0.4% were rated no response
- 0.2% were not rated
Study design: Children (n=27) <6 years of age with severe or moderately severe hemophilia A (FVIII:C ≤2% and with at least 20 prior exposure days) received XYNTHA for on-demand and follow-up treatment. Results reported here for 24 patients (493 bleeding episodes). The median dose/infusion was 28 IU/kg.
In comparison to the pharmacokinetic parameters reported in adults, children have shorter half-lives, larger volumes of distribution, and lower recovery of factor VIII after XYNTHA administration. Larger or more frequent doses may be required to account for the observed differences in pharmacokinetic parameters.
Demonstrated in clinical studies to have a low inhibitor rate
The development of factor VIII inhibitors was evaluated in 5 clinical studies of 144 adult and pediatric previously treated patients with ≥50 exposure days:
- Across all studies, 2 adult and 1 pediatric PTPs (2.1%) developed FVIII inhibitors
- In the surgical study of 30 patients, one low-titer, persistent inhibitor and one transient, false-positive inhibitor were without significance
- Results were not clinically significant and these patients did not require treatment
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