Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study
| العنوان: | Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study |
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| المؤلفون: | Maria Elisa Mancuso, Carmen Altisent, Hervé Chambost, M. Jansen, Anthony K.C. Chan, Mark Belletrutti, Jonathan M. Ducore, Paolo Gresele, John K. Wu, Ri Liesner, Sunil Lohade, Manuela Carvalho, Berardino Pollio, Benoît Guillet, Aby Abraham, Johannes Oldenburg, Sigurd Knaub, Anna Klukowska, Ellis J. Neufeld, Anna Pavlova, Leonid Dubey, Marianne Sigaud, Manuel Carcao, Víctor Jiménez-Yuste, Lidija Kitanovski, Larisa Belyanskaya, Kateryna Vilchevska, Michael Gattens, Olaf Walter, Vladimir Vdovin, Yves Gruel |
| المساهمون: | Great Ormond Street Hospital for Children [London] (GOSH), Vall d'Hebron University Hospital [Barcelona], University of Alberta, The Hospital for sick children [Toronto] (SickKids), Hospital de São João [Porto], Centre recherche en CardioVasculaire et Nutrition = Center for CardioVascular and Nutrition research (C2VN), Aix Marseille Université (AMU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE), McMaster University [Hamilton, Ontario], University of California [Davis] (UC Davis), University of California, Cambridge University Hospitals - NHS (CUH), University of Cambridge [UK] (CAM), Università degli Studi di Perugia (UNIPG), CHU Trousseau [APHP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Institut de recherche en santé, environnement et travail (Irset), Université d'Angers (UA)-Université de Rennes 1 (UR1), Université de Rennes (UNIV-RENNES)-Université de Rennes (UNIV-RENNES)-École des Hautes Études en Santé Publique [EHESP] (EHESP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Structure Fédérative de Recherche en Biologie et Santé de Rennes ( Biosit : Biologie - Santé - Innovation Technologique ), Universidad Autonoma de Madrid (UAM), University Medical Center, Medical University of Warsaw - Poland, Istituti di Ricovero e Cura a Carattere Scientifico (IRCCS), Universitätsklinikum Bonn (UKB), Centre hospitalier universitaire de Nantes (CHU Nantes), BC Children's Hospital Research Institute [Vancouver, BC, Canada] (BCCHR), University of British Columbia (UBC), St Jude Children's Research Hospital, Octapharma, University of California (UC), Università degli Studi di Perugia = University of Perugia (UNIPG), Université d'Angers (UA)-Université de Rennes (UR)-École des Hautes Études en Santé Publique [EHESP] (EHESP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Structure Fédérative de Recherche en Biologie et Santé de Rennes ( Biosit : Biologie - Santé - Innovation Technologique ), Universidad Autónoma de Madrid (UAM) |
| المصدر: | Thrombosis and Haemostasis Thrombosis and Haemostasis, Schattauer, 2021, ⟨10.1055/s-0040-1722623⟩ Thrombosis and Haemostasis, 2021, 121 (11), pp.1400-1408. ⟨10.1055/s-0040-1722623⟩ |
| بيانات النشر: | HAL CCSD, 2021. |
| سنة النشر: | 2021 |
| مصطلحات موضوعية: | Male, medicine.medical_specialty, Time Factors, Haemophilia A, haemophilia, Hemorrhage, 030204 cardiovascular system & hematology, Hemophilia A, Haemophilia, Severity of Illness Index, Gastroenterology, Antibodies, 03 medical and health sciences, 0302 clinical medicine, Interquartile range, Internal medicine, hemic and lymphatic diseases, medicine, Humans, Genetic Predisposition to Disease, Prospective Studies, coagulation, Factor VIII, Coagulants, business.industry, Immunogenicity, Infant, FVIII inhibitors, [SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology, Hematology, medicine.disease, Recombinant Proteins, 3. Good health, Clinical trial, Titer, Treatment Outcome, Coagulation, Mutation, Complication, business, Coagulation and Fibrinolysis, 030215 immunology |
| الوصف: | Introduction FVIII inhibitor development is the most serious contemporary treatment complication in haemophilia A, particularly in previously untreated patients (PUPs). No inhibitors developed in clinical trials in previously treated patients treated with simoctocog alfa (Nuwiq), a fourth-generation recombinant FVIII produced in a human cell line. Methods The NuProtect study investigated the immunogenicity of simoctocog alfa in PUPs. NuProtect was a prospective, multinational, open-label, non-controlled, phase III study. PUPs with severe haemophilia A (FVIII:C Results A total of 108 PUPs with a median age at first treatment of 12.0 months (interquartile range: 8.0–23.5) were treated with simoctocog alfa. F8 mutation type was known for 102 patients (94.4%) of whom 90 (88.2%) had null F8 mutations and 12 (11.8%) had non-null mutations. Of 105 PUPs evaluable for inhibitor development, 28 (26.7%) developed inhibitors; 17 high titre (16.2%) and 11 low titre (10.5%). No PUPs with non-null F8 mutations developed inhibitors. Conclusion In the NuProtect study, the rate of inhibitor development in PUPs with severe haemophilia A treated with simoctocog alfa was lower than the rate reported for hamster-cell-derived recombinant factor VIII products in other recent clinical trials. No inhibitors were reported in PUPs with non-null F8 mutations. |
| اللغة: | English |
| تدمد: | 0340-6245 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8796168da5f744cf29cd3b770616d144 https://hal.archives-ouvertes.fr/hal-03162369/document |
| حقوق: | OPEN |
| رقم الأكسشن: | edsair.doi.dedup.....8796168da5f744cf29cd3b770616d144 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 03406245 |
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