Abstract Number: PB0999
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: The NuProtect (GENA-05) study assessed the immunogenicity, efficacy and safety of simoctocog alfa in 108 previously untreated patients (PUPs) with severe haemophilia A. The cumulative incidence of high-titre inhibitors was 17.6%.
Aims: The key objectives of the GENA-15 study were to investigate the long-term immunogenicity, efficacy and safety of simoctocog alfa in patients who had completed the NuProtect study.
Methods: GENA-15 was a prospective, multicentre, multinational, open-label, non-controlled phase 3b extension study. Participation was offered to all patients who completed the NuProtect study and had been treated for at least 100 exposure days (EDs). GENA-15 was conducted in accordance with the ethical principles of the Declaration of Helsinki.
Results: Forty-eight patients were enrolled into GENA-15. All patients were included in the safety population. Efficacy data were available for 47 patients, all of whom received prophylaxis. Approximately 5 million international units (IU) of simoctocog alfa were administered to 48 patients with a mean of 179 EDs, a total of 8603 EDs, and a total of 8646 infusions. No patient developed FVIII inhibitors during GENA-15. The annualised bleeding rates (ABRs) during prophylaxis (negative binomial counting regression model) were 0.28 (95% confidence interval [CI]: 0.15, 0.53) [median 0, range 0-3.88] for spontaneous bleeding episodes (BEs) and 1.62 (95% CI: 1.09, 2.42) [median 1.0, range 0-8.68] for all BEs. Of 111 BEs treated with simoctocog alfa in 29 patients, efficacy of treatment was rated as “excellent” or “good” for 86 (77.5%). For two major surgeries (in two patients) the overall efficacy assessment was excellent. There were no adverse events assessed as related to study treatment by the Investigator.
Conclusions: No FVIII inhibitors developed during long-term simoctocog alfa prophylaxis in patients with severe haemophilia A who had completed the NuProtect PUP study. Simoctocog alfa prophylaxis was effective and well tolerated.
To cite this abstract in AMA style:
Liesner RJ, GENA-15 Study Group . Long-Term Immunogenicity, Efficacy and Safety of Simoctocog Alfa in Patients with Severe Haemophilia A who Had Completed the NuProtect PUP Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/long-term-immunogenicity-efficacy-and-safety-of-simoctocog-alfa-in-patients-with-severe-haemophilia-a-who-had-completed-the-nuprotect-pup-study/. Accessed March 21, 2024.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/long-term-immunogenicity-efficacy-and-safety-of-simoctocog-alfa-in-patients-with-severe-haemophilia-a-who-had-completed-the-nuprotect-pup-study/