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

Autor: Mathias M; Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children NHS Trust Haemophilia Centre, NIHR GOSH BRC, London, UK., Abraham A; Department of Hematology, Christian Medical College, Vellore, India., Belletrutti MJ; Department of Pediatrics, Division of Hematology/Oncology/BMT, University of British Columbia and British Columbia Children's Hospital, Vancouver, Canada., Carcao M; Department of Paediatrics, Division of Haematology/Oncology and Child Health Evaluative Sciences, Research Institute Hospital for Sick Children, Toronto, Canada., Carvalho M; Congenital Coagulopathies Reference Centre, São João University Hospital Centre, Porto, Portugal., Chambost H; AP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone, Aix Marseille Univ INSERM, INRA, C2VN, Marseille, France., Chan AKC; Department of Pediatrics, McMaster Centre of Transfusion Research, McMaster University, Hamilton, Canada., Dubey L; Department of Paediatrics, Western Ukrainian Specialized Children's Medical Centre, Lviv, Ukraine., Ducore J; Department of Pediatrics, University of California Davis Medical Center, Sacramento, California, USA., Gattens M; Department of Paediatric Haematology and Oncology, Addenbrooke's Hospital, Cambridge University Hospital NHS Foundation Trust, Cambridge, UK., Gresele P; Department of Medicine and Surgery, University of Perugia, Perugia, Italy., Gruel Y; Centre Régional de Traitement de l'Hémophilie, Hôpital Trousseau, Tours, France., Guillet B; Haemophilia Treatment Centre, Univ Rennes, CHU Rennes, INSERM, EHESP, Irset (Institut de Recherche en Santé, Environnement et Travail) - UMR_S 1085, Rennes, France., Jiménez-Yuste V; Servicio de Hematología, Hospital Univeristario La Paz, Autónoma, University of Madrid, Madrid, Spain., Kitanovski L; Department of Haematooncology, Division of Paediatrics, University Medical Center Ljubljana, Ljubljana, Slovenia., Klukowska A; Haemostasis Group of the Polish Society of Haematology and Transfusiology, Warsaw, Poland., Lohade S; Department of Hematology, Sahyadri Speciality Hospital, Pune, India., Mancuso ME; Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Milan, Italy., Oldenburg J; Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany., Pollio B; Regional Reference Centre for Inherited Bleeding and Thrombotic Disorders, Regina Margherita Children Hospital, Turin, Italy., Sigaud M; Centre Régional de Traitement de I'Hémophilie, University Hospital of Nantes, Nantes, France., Vilchevska K; Department of Hematology, OHMATDYT - National Specialized Children's Hospital, Kiev, Ukraine., Wu JKM; Department of Pediatrics, Division of Hematology/Oncology/BMT, University of British Columbia and British Columbia Children's Hospital, Vancouver, Canada., Jansen M; Octapharma Pharmazeutika Produktionsges m.b.H, Vienna, Austria., Belyanskaya L; Octapharma AG, Lachen, Switzerland., Walter O; Octapharma AG, Lachen, Switzerland., Knaub S; Octapharma AG, Lachen, Switzerland., Neufeld EJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Jazyk: angličtina
Zdroj: European journal of haematology [Eur J Haematol] 2023 Oct; Vol. 111 (4), pp. 544-552. Date of Electronic Publication: 2023 Jul 13.
DOI: 10.1111/ejh.14040
Abstrakt: 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.
(© 2023 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd.)
Databáze: MEDLINE
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