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Liesner RJ, Abraham A, Altisent C, et al. Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study. Thromb Haemost. 2021;121(11):1400-1408doi: 10.1055/s-0040-1722623.
Liesner, R. J., Abraham, A., Altisent, C., Belletrutti, M. J., Carcao, M., Carvalho, M., Chambost, H., Chan, A. K. C., Dubey, L., Ducore, J., Gattens, M., Gresele, P., Gruel, Y., Guillet, B., Jimenez-Yuste, V., Kitanovski, L., Klukowska, A., Lohade, S., Mancuso, M. E., Oldenburg, J., Pavlova, A., Pollio, B., Sigaud, M., Vdovin, V., Vilchevska, K., Wu, J. K. M., Jansen, M., Belyanskaya, L., Walter, O., Knaub, S., & Neufeld, E. J. (2021). Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study. Thrombosis and haemostasis, 121(11), 1400-1408. https://doi.org/10.1055/s-0040-1722623
Liesner, Ri J, et al. "Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study." Thrombosis and haemostasis vol. 121,11 (2021): 1400-1408. doi: https://doi.org/10.1055/s-0040-1722623
Liesner RJ, Abraham A, Altisent C, Belletrutti MJ, Carcao M, Carvalho M, Chambost H, Chan AKC, Dubey L, Ducore J, Gattens M, Gresele P, Gruel Y, Guillet B, Jimenez-Yuste V, Kitanovski L, Klukowska A, Lohade S, Mancuso ME, Oldenburg J, Pavlova A, Pollio B, Sigaud M, Vdovin V, Vilchevska K, Wu JKM, Jansen M, Belyanskaya L, Walter O, Knaub S, Neufeld EJ. Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study. Thromb Haemost. 2021 Nov;121(11):1400-1408. doi: 10.1055/s-0040-1722623. Epub 2021 Feb 13. PMID: 33581698; PMCID: PMC8570909.
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Thromb Haemost. 2021 Nov;121(11):1400-1408. doi: 10.1055/s-0040-1722623. Epub 2021 Feb 13.

Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study.

Thrombosis and haemostasis

Ri J Liesner, Aby Abraham, Carmen Altisent, Mark J Belletrutti, Manuel Carcao, Manuela Carvalho, Hervé Chambost, Anthony K C Chan, Leonid Dubey, Jonathan Ducore, Michael Gattens, Paolo Gresele, Yves Gruel, Benoit Guillet, Victor Jimenez-Yuste, Lidija Kitanovski, Anna Klukowska, Sunil Lohade, Maria Elisa Mancuso, Johannes Oldenburg, Anna Pavlova, Berardino Pollio, Marianne Sigaud, Vladimir Vdovin, Kateryna Vilchevska, John K M Wu, Martina Jansen, Larisa Belyanskaya, Olaf Walter, Sigurd Knaub, Ellis J Neufeld

Affiliations

  1. Great Ormond Street Hospital for Children NHS Trust Haemophilia Centre, NIHR GOSH BRC, London, United Kingdom.
  2. Department of Hematology, Christian Medical College, Vellore, India.
  3. Unitat d'Hemofilia, Hospital Vall D'Hebron, Barcelona, Spain.
  4. Pediatric Hematology, Department of Pediatrics, University of Alberta, Edmonton, Canada.
  5. Division of Haematology/Oncology and Child Health Evaluative Sciences, Department of Paediatrics, Research Institute, Hospital for Sick Children, Toronto, Canada.
  6. Congenital Coagulopathies Reference Centre, São João University Hospital Centre, Porto, Portugal.
  7. AP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone, Aix Marseille Univ, INSERM, INRA, C2VN, Marseille, France.
  8. Division of Pediatric Hematology/Oncology, McMaster University, Hamilton, Canada.
  9. Department of Pediatrics, Western Ukrainian Specialized Children's Medical Centre, Lviv, Ukraine.
  10. Department of Pediatrics, University of California Davis Medical Center, Sacramento, United States.
  11. Department of Paediatric Haematology and Oncology, Addenbrooke's Hospital, Cambridge University Hospital NHS Foundation Trust, Cambridge, United Kingdom.
  12. Department of Medicine and Surgery, University of Perugia, Perugia, Italy.
  13. Centre Régional de Traitement de l'Hémophilie, Hôpital Trousseau, Tours, France.
  14. Haemophilia Treatment Centre, Univ Rennes, CHU Rennes, Inserm, EHESP, Irset (Institut de recherche en santé, environnement et travail) - UMR_S 1085, Rennes, France.
  15. Servicio de Hematología, Hospital Univeristario La Paz, Autónoma University, Madrid, Spain.
  16. Department of Haemato-Oncology, University Medical Center Ljubljana, Ljubljana, Slovenia.
  17. Department of Pediatrics, Haematology and Oncology, Warsaw Medical University, Warsaw, Poland.
  18. Department of Hematology, Sahyadri Speciality Hospital, Pune, India.
  19. Center for Thrombosis and Hemorrhagic Diseases, Humanitas Clinical and Research Center - IRCCS, Rozzano, Milan, Italy.
  20. Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany.
  21. Department of Transfusion Medicine, Regina Margherita Children Hospital of Turin, Turin, Italy.
  22. Centre Régional de Traitement de I'Hémophilie, University Hospital of Nantes, Nantes, France.
  23. Department of Hematology, Morozovskaya Children's Hospital, Moscow, Russian Federation.
  24. Department of Hematology, State Institution "Institute of Urgent and Reconstructive Surgery named after V.K. Gusak of National Academy of Medical Sciences of Ukraine," Donetsk, Ukraine.
  25. British Columbia Children's Hospital, Vancouver, Canada.
  26. Octapharma Pharmazeutika Produktionsges.mbH, Vienna, Austria.
  27. Octapharma AG, Lachen, Switzerland.
  28. St. Jude Children's Research Hospital, Memphis, Tennessee, United States.

PMID: 33581698 PMCID: PMC8570909 DOI: 10.1055/s-0040-1722623

Abstract

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 <1%) of any age and ethnicity were treated with simoctocog alfa for 100 exposure days or a maximum of 5 years. Patients were true PUPs without prior exposure to FVIII concentrates or blood components. Inhibitor titres were measured with the Nijmegen-modified Bethesda assay; cut-off for positivity was 0.6 BU mL

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.

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

The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution License, permitting unrestricted use, distribution, and reproduction so long as the original work is properly cited. (https://creativecommons.org/licenses/by/4.0/).

Conflict of interest statement

R. J. Liesner, A. Abraham, C. Altisent, M. J. Belletrutti, M. Carcao, M. Carvalho, H. Chambost, A. K. C. Chan, L. Dubey, J. Ducore, M. Gattens, P. Gresele, Y. Gruel, B. Guillet, V. J. Yuste, L. Kitano

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