Abstract Number: PB0590
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Inhibitors are the most serious treatment-related complication of haemophilia A and develop in up to 40% of previously untreated patients (PUPs). The NuProtect study assessed the immunogenicity, efficacy and safety of simoctocog alfa, a 4th generation recombinant FVIII produced in a human cell line, in 108 PUPs with haemophilia A. High-titre inhibitors developed in 16% of patients. NuProtect is the largest prospective study of a single FVIII product in true PUPs to date.
Aims: To assess the efficacy of simoctocog alfa for the prevention and treatment of bleeding in PUPs.
Methods: NuProtect was a prospective, multinational, open-label, non-controlled phase III study of 108 PUPs of any age and ethnicity with severe haemophilia A. Patients received simoctocog alfa for 100 exposure days or up to 5 years for prophylaxis, surgical prophylaxis or on-demand treatment. Informed consent was obtained from patients/caregivers and the study was approved by all relevant independent ethics committees and institutional review boards.
Results: In PUPs who received continuous prophylaxis for ≥24 weeks (n=50), the mean (SD) annualised bleeding rate (ABR) was 0.54 (1.07) for spontaneous bleeding episodes (BEs) and 3.61 (3.82) for all BEs. The efficacy of continuous prophylaxis was rated as excellent or good in 100% of patients for spontaneous BEs and 96% for all BEs. A total of 808 BEs in 85 patients were treated during inhibitor-free periods, of which 502 (62.1%) occurred in 51 patients during on-demand treatment. Efficacy in treating BEs was rated as excellent or good in 92.9% of 804 rated BEs. The majority of BEs (92.3%) were controlled with 1 or 2 infusions. Efficacy in surgical prophylaxis was rated as excellent or good in 94.7% (18/19) of procedures.
Conclusions: Simoctocog alfa was efficacious for the prevention and treatment of BEs in PUPs with haemophilia A, including during surgery.
To cite this abstract in AMA style:
Neufeld EJ, Liesner RJ. Efficacy of Simoctocog Alfa in Previously Untreated Patients with Severe Haemophilia A: Final Results from the NuProtect Study [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/efficacy-of-simoctocog-alfa-in-previously-untreated-patients-with-severe-haemophilia-a-final-results-from-the-nuprotect-study/. Accessed March 22, 2024.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/efficacy-of-simoctocog-alfa-in-previously-untreated-patients-with-severe-haemophilia-a-final-results-from-the-nuprotect-study/