Simoctocog alfa (Nuwiq®) in previously untreated patients with severe haemophilia A—Final efficacy and safety results from the NuProtect study - Université de Rennes Accéder directement au contenu
Article Dans Une Revue European Journal of Haematology Année : 2023

Simoctocog alfa (Nuwiq®) in previously untreated patients with severe haemophilia A—Final efficacy and safety results from the NuProtect study

Mary Mathias
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Leonid Dubey
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Jonathan Ducore
Yves Gruel
Victor Jiménez-Yuste
Anna Klukowska
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Sunil Lohade
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Martina Jansen
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Larisa Belyanskaya
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Olaf Walter
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Sigurd Knaub
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Résumé

Introduction: Simoctocog alfa (Nuwiq®) is a 4th generation recombinant FVIII with proven efficacy for the prevention and treatment of bleeding episodes (BEs) in previously treated patients with severe haemophilia A. The NuProtect study assessed the immunogenicity, efficacy and safety of simoctocog alfa in 108 previously untreated patients (PUPs). The incidence of high-titre inhibitors was 16.2% and no patients with non-null F8 mutations developed inhibitors.Aim: To report the efficacy and safety results from the NuProtect study.Methods: PUPs received simoctocog alfa for prophylaxis, treatment of BEs, or as surgical prophylaxis. The efficacy of prophylaxis (during inhibitor-free periods) was assessed using annualised bleeding rates (ABRs). The efficacy in treating BEs and in surgical prophylaxis was assessed using a 4-point scale. Adverse events were recorded throughout the study.Results: Of 108 PUPs treated with simoctocog alfa, 103 received at least one prophylactic dose and 50 received continuous prophylaxis for at least 24 weeks. In patients on continuous prophylaxis, the median ABR was 0 (mean 0.5) for spontaneous BEs and 2.5 (mean 3.6) for all BEs. In 85 patients who had BEs, efficacy of BE treatment was excellent or good for 92.9% (747/804) of rated BEs; 92.3% of BEs were treated with 1 or 2 infusions. The efficacy of surgical prophylaxis was excellent or good for 94.7% (18/19) of rated procedures. There were no safety concerns and no thromboembolic events.Conclusion: Simoctocog alfa was efficacious and well tolerated as prophylaxis, surgical prophylaxis and for the treatment of BEs in PUPs with severe haemophilia A.

Domaines

Hématologie

Dates et versions

hal-04165547 , version 1 (19-07-2023)

Licence

Paternité - Pas d'utilisation commerciale - Pas de modification

Identifiants

Citer

Mary Mathias, Aby Abraham, Mark Belletrutti, Manuel Carcao, Manuela Carvalho, et al.. Simoctocog alfa (Nuwiq®) in previously untreated patients with severe haemophilia A—Final efficacy and safety results from the NuProtect study. European Journal of Haematology, 2023, 111 (4), pp.544-552. ⟨10.1111/ejh.14040⟩. ⟨hal-04165547⟩
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