Abstract Number: PB0535
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: In children with severe haemophilia A, FVIII prophylaxis prevents life-threatening bleeds, protects from joint damage and enables daily activities. Simoctocog alfa (Nuwiq®) has proven efficacy and safety in the prevention and treatment of bleeds in children and adults, and is associated with a low rate of inhibitor development in previously untreated patients.
Aims: To assess the efficacy and safety of long-term prophylaxis with simoctocog alfa in children.
Methods: This is a pooled analysis of children with severe haemophilia A enrolled in the prospective GENA-15 and GENA-13 studies. GENA-15 and GENA-13 were long-term extension studies of the GENA-05 (NuProtect) and GENA-03 studies in previously untreated and previously treated children, respectively. Prophylaxis duration was defined as the time from first until last prophylactic dose.
Results: 97 children were enrolled in the extension studies and 96 were included in these analyses. One child who had undergone immune tolerance induction was excluded. The median (IQR) age at prophylaxis start was 3 (1–6) years, and the median (IQR) duration of prophylaxis was 359 (300–509) exposure days over 36.4 (31.6–40.9) months. The median (IQR) annualised bleeding rate (ABR) was 0.31 (0–1.08) for spontaneous bleeds and 1.81 (0.58–3.94) for all bleeds; 44.8% of patients experienced no spontaneous bleeds over a median of 3 years. A total of 763 bleeds were treated with simoctocog alfa. Most (86.5%) were managed with 1 or 2 infusions, and 85.7% (649/757) of rated bleeds were treated successfully (excellent or good). None of the 96 patients on long-term prophylaxis withdrew from the studies due to an adverse event and there were no thrombotic events.
Conclusions: Simoctocog alfa is efficacious and well-tolerated for long-term prophylaxis in children with severe haemophilia A and is an attractive long-term therapeutic option for children.
To cite this abstract in AMA style:
Klukowska A, Jansen M, Knaub S, Liesner RJ. Long-term Prophylaxis with Simoctocog Alfa in Children [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/long-term-prophylaxis-with-simoctocog-alfa-in-children/. Accessed September 27, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/long-term-prophylaxis-with-simoctocog-alfa-in-children/