Clinical experience with moroctocog alfa (AF‐CC) in younger paediatric patients with severe haemophilia A: Two open‐label studies. Issue 4 (26th March 2018) - Rusen, L.
Abstract : Introduction: The pharmacokinetics (PK), efficacy and safety of moroctocog alfa (AF‐CC) have been demonstrated in haemophilia A patients aged ≥6 years. Aim: These studies aimed to further describe moroctocog alfa (AF‐CC) experience in paediatric patients (<12 years) with severe haemophilia A (FVIII:C < 1%). Methods: Two prospective, open‐label studies enrolled patients aged <12 years: one study with 37 previously treated patients (PTPs) and another with 23 previously untreated patients (PUPs). All patients initially received 50 IU/kg of moroctocog alfa (AF‐CC) to evaluate either recovery alone, or with other PK parameters (6 to <12 years) before continuing treatment for 100 exposure days (EDs) or 24 months. Results: At baseline, mean (±SD) recovery ranged between 1.32 ± 0.65 (PUPs aged <2 years) and 2.13 ± 0.82 (PTPs aged 6 to <12 years). The mean (±SD) half‐life was 9.12 ± 1.94 hours in PTPs aged 6 to <12 years. No new safety signals were detected in either study, 2 transient lower titre inhibitors occurred in PTPs while 8 inhibitors (3 low and 5 high titre) were detected in PUPs. Most bleeding episodes resolved with one infusion (94% [893/954]). The annualised bleeding rate (ABR) in the PTP study was 27.5 and 4.2 for patients reporting an on‐demand and routine prophylaxis regimen at baseline, respectively. In the PUP study, the overall ABR was 5.9. Conclusion: Moroctocog alfa (AF‐CC) had expected PK findings (lower recovery in young children compared with older children) along with being safe and efficacious in a population of young severe haemophilia A patients.