{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T22:23:57Z","timestamp":1790634237944,"version":"4.1.0"},"reference-count":26,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2023,7,13]],"date-time":"2023-07-13T00:00:00Z","timestamp":1689206400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100016300","name":"Octapharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100016300","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["European J of Haematology"],"published-print":{"date-parts":[[2023,10]]},"abstract":"Abstract<\/jats:title>Introduction<\/jats:title>Simoctocog alfa (Nuwiq\u00ae) 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\u2010titre inhibitors was 16.2% and no patients with non\u2010null F8<\/jats:italic> mutations developed inhibitors.<\/jats:p><\/jats:sec>Aim<\/jats:title>To report the efficacy and safety results from the NuProtect study.<\/jats:p><\/jats:sec>Methods<\/jats:title>PUPs received simoctocog alfa for prophylaxis, treatment of BEs, or as surgical prophylaxis. The efficacy of prophylaxis (during inhibitor\u2010free periods) was assessed using annualised bleeding rates (ABRs). The efficacy in treating BEs and in surgical prophylaxis was assessed using a 4\u2010point scale. Adverse events were recorded throughout the study.<\/jats:p><\/jats:sec>Results<\/jats:title>Of 108 PUPs treated with simoctocog alfa, 103 received at least one prophylactic dose and 50 received continuous prophylaxis for at least 24\u2009weeks. 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.<\/jats:p><\/jats:sec>Conclusion<\/jats:title>Simoctocog alfa was efficacious and well tolerated as prophylaxis, surgical prophylaxis and for the treatment of BEs in PUPs with severe haemophilia A.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.14040","type":"journal-article","created":{"date-parts":[[2023,7,13]],"date-time":"2023-07-13T07:44:56Z","timestamp":1689234296000},"page":"544-552","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Simoctocog alfa (Nuwiq\u00ae) in previously untreated patients with severe haemophilia A\u2014Final efficacy and safety results from the NuProtect<\/scp> study"],"prefix":"10.1111","volume":"111","author":[{"given":"Mary","family":"Mathias","sequence":"first","affiliation":[{"name":"Haemophilia Comprehensive Care Centre Great Ormond Street Hospital for Children NHS Trust Haemophilia Centre, NIHR GOSH BRC London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aby","family":"Abraham","sequence":"additional","affiliation":[{"name":"Department of Hematology Christian Medical College Vellore India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark J.","family":"Belletrutti","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Hematology\/Oncology\/BMT University of British Columbia and British Columbia Children's Hospital Vancouver Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manuel","family":"Carcao","sequence":"additional","affiliation":[{"name":"Department of Paediatrics, Division of Haematology\/Oncology and Child Health Evaluative Sciences Research Institute Hospital for Sick Children Toronto Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manuela","family":"Carvalho","sequence":"additional","affiliation":[{"name":"Congenital Coagulopathies Reference Centre S\u00e3o Jo\u00e3o University Hospital Centre Porto Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Herv\u00e9","family":"Chambost","sequence":"additional","affiliation":[{"name":"AP\u2010HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone Aix Marseille Univ INSERM, INRA, C2VN Marseille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anthony K. 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