Abstract Number: PB1149
Meeting: ISTH 2022 Congress
Background: Simoctocog alfa (Nuwiq®) is a human cell line-derived recombinant human FVIII that was developed with the aim to provide a low immunogenicity product with excellent haemostatic efficacy for people with haemophilia A. The efficacy and safety of Nuwiq® have been demonstrated in clinical trials of previously treated patients (PTPs) and previously untreated patients with severe haemophilia A.
Aims: To assess the long-term immunogenicity, safety and efficacy of Nuwiq® in a pooled analysis of PTPs with haemophilia A.
Methods: Data from patients who received Nuwiq® prophylaxis for ≥100 exposure days in two observational and four interventional post-authorisation studies were pooled. These studies included male PTPs with severe haemophilia A (FVIII:C < 1%); one study included patients with FVIII:C ≤2%. Cut-off dates were used for studies ongoing at the time of database lock.
Results: Demographics of the 216 patients included in the analysis are shown in Table 1. There were no reports of inhibitor development in any patient. Twelve adverse drug reactions occurred in 8 patients; one was serious (mild pyrexia requiring hospitalisation) but resolved and treatment was continued. No thromboembolic events occurred. The median (interquartile range [IQR]) duration of prophylaxis was 16.8 months (9.4–24.4); 108 (50.0%) patients experienced no spontaneous bleeding episodes (BEs) during prophylaxis. Median (IQR) annualised bleeding rates were 0.11 (0–1.45), 0.50 (0–1.95) and 1.78 (0–4.24) for spontaneous, traumatic and all BEs, respectively. A total of 1011 BEs in 152 patients were treated with Nuwiq®. Most BEs (860 [85.1%]) resolved with ≤2 infusions of Nuwiq®; treatment efficacy was rated excellent or good for the majority (84.3%) of BEs. Of 36 surgeries with a postoperative rating by a haematologist, 31 (86.1%) were rated successful.
Conclusion(s): These pooled long-term data from PTPs confirm the safety and efficacy of Nuwiq® demonstrated previously in clinical trials.
To cite this abstract in AMA style:Mathias M, Borchiellini A, Peyvandi F, Chistolini A, Marino R, Rocino A, Sol Cruz M, Carolina Oliva S, Molina Pantoja A, Grimley C, Bayart S, Klukowska A, Lissitchkov T, Halimeh S, Werner S, Pezeshki G, Knaub S, Kessler C. Pooled analysis of long-term efficacy and safety of simoctocog alfa in previously treated patients with haemophilia A [abstract]. https://abstracts.isth.org/abstract/pooled-analysis-of-long-term-efficacy-and-safety-of-simoctocog-alfa-in-previously-treated-patients-with-haemophilia-a/. Accessed October 1, 2023.
« Back to ISTH 2022 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/pooled-analysis-of-long-term-efficacy-and-safety-of-simoctocog-alfa-in-previously-treated-patients-with-haemophilia-a/