Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A : Final Results of the NuProtect Study
Liesner, Ri J. (Great Ormond Street Hospital for Children (Londres))
Abraham, Aby (Christian Medical College (Vellore, India). Department of Hematology)
Altisent, Carmen (Hospital Universitari Vall d'Hebron)
Belletrutti, Mark J. (University of Alberta. Department of Pediatrics)
Carcao, Manuel (Hospital for Sick Children (Toronto, Canada). Department of Paediatrics)
Carvalho, Manuela (São João University Hospital Centre (Porto, Portugal))
Chambost, Hervé (Aix Marseille University. Department of Pediatric Hematology Oncology)
Chan, Anthony K. C. (McMaster University. Division of Pediatric Hematology/Oncology)
Dubey, Leonid (Western Ukrainian Specialized Children's Medical Centre. Department of Pediatrics)
Ducore, Jonathan (University of California Davis Medical Center. Department of Pediatrics)
Gattens, Michael (Cambridge University Hospitals NHS Foundation Trust (Regne Unit))
Gresele, Paolo (University of Perugia. Department of Medicine and Surgery)
Gruel, Yves (Hôpital Trousseau (Tours, França))
Guillet, Benoit (Univ Rennes. Haemophilia Treatment Centre)
Jimenez-Yuste, Victor (Hospital Universitario La Paz (Madrid))
Kitanovski, Lidija (University Medical Center Ljubljana. Department of Haemato-Oncology)
Klukowska, Anna (Warsaw Medical University. Department of Pediatrics, Haematology and Oncology)
Lohade, Sunil (Sahyadri Speciality Hospital. Department of Hematology)
Mancuso, Maria Elisa (Humanitas Clinical and Research Center)
Oldenburg, Johannes (University Clinic Bonn. Institute of Experimental Haematology and Transfusion Medicine)
Pavlova, Anna (University Clinic Bonn. Institute of Experimental Haematology and Transfusion Medicine)
Pollio, Berardino (Regina Margherita Children Hospital of Turin. Department of Transfusion Medicine)
Sigaud, Marianne (CHU de Nantes)
Vdovin, Vladimir (Morozovskaya Children's Hospital. Department of Hematology)
Vilchevska, Kateryna (Institute of Urgent and Reconstructive Surgery named after V.K. Gusak of National Academy of Medical Sciences of Ukraine.Department of Hematology)
Wu, John K. M. (British Columbia Children's Hospital (Vancouver, Canadà))
Jansen, Martina (Octapharma Pharmazeutika Produktionsges.mbH)
Belyanskaya, Larisa (Octapharma AG)
Walter, Olaf (Octapharma AG)
Knaub, Sigurd (Octapharma AG)
Neufeld, Ellis J. (St. Jude Children's Research Hospital (Memphis, Estats Units d'Amèrica))

Data:	2021
Resum:	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 −1 (≥0. 6 to <5 low-titre, ≥5 high titre). 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.
Nota:	Funding: This trial was sponsored by Octapharma AG (Lachen, Switzerland)
Drets:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Llengua:	Anglès
Document:	Estudi clínic ; recerca ; Versió publicada
Matèria:	Coagulation ; FVIII inhibitors ; Haemophilia
Publicat a:	Thrombosis and Haemostasis, Vol. 121 Núm. 11 (february 2021) , p. 1400-1408, ISSN 2567-689X

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

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