Summary
The Phase 3 A-LONG and Kids A-LONG studies demonstrated the prolonged half-life of
rFVIIIFc compared with rFVIII, and the safety and efficacy of rFVIIIFc in subjects
with severe haemophilia A. Eligible subjects from A-LONG and Kids A-LONG continued
rFVIIIFc treatment by enrolling in ASPIRE, an ongoing extension study. Based on combined
data from the primary studies and ASPIRE interim data, the safety and efficacy of
rFVIIIFc in subjects requiring surgery were evaluated. Perioperative dosing regimens
were determined by investigators with guidance based on pharmacokinetic data and recommendations
from a clinical dosing committee. In addition to dosing frequency, factor consumption,
blood loss, transfusions, bleeding episodes, and haemostatic response were assessed.
Across studies, 21 subjects underwent 23 evaluable major surgeries, including 19 orthopaedic
surgeries; 41 subjects underwent 52 minor surgeries, including 30 dental procedures. No major and 10 minor surgeries were performed in paediatric subjects. Of the major (n = 22) and minor
(n = 32) surgeries assessed for haemostatic response, all were rated as excellent
or good by the investigator/surgeon. During most major surgeries (95.7 %), haemostasis
was maintained with one rFVIIIFc infusion. Blood loss in major surgeries was consistent
with similar surgeries in subjects without haemophilia. Across studies, rFVIIIFc was
well tolerated; no subject developed an inhibitor.
Keywords
Phase 3 trial - factor VIII - haemophilia A - haemostasis - surgery
