{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T15:29:19Z","timestamp":1790868559508,"version":"4.1.0"},"reference-count":26,"publisher":"Georg Thieme Verlag KG","issue":"09","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Thromb Haemost"],"published-print":{"date-parts":[[2017]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>Turoctocog alfa pegol (N8-GP, Novo Nordisk, Bagsv\u00e6rd, Denmark), an extended half-life glycoPEGylated recombinant factor VIII (rFVIII), is being developed for prophylaxis and treatment of bleeds in haemophilia A patients. pathfinder\u21225 is a multinational, open-label, single-arm trial to assess safety, efficacy and pharmacokinetics of N8-GP in paediatric (&lt;12 years), previously treated patients. Boys with severe haemophilia A (&lt;1 % FVIII), no history of inhibitors and previously treated with FVIII products (&gt;50 exposure days [ED] for patients aged 0\u20135 years [younger cohort]; &gt;150 ED for patients aged 6\u201311 years [older cohort]) were included. For prophylaxis, N8-GP was dosed at 50\u201375 IU\/kg twice weekly; bleeds were treated with 20\u201375 IU\/kg. Half-life was estimated for the patients\u2019 previous FVIII product and for N8-GP. Sixty-eight patients received N8-GP; none developed inhibitors and no other concerns were identified. Median annualised bleeding rate was 1.95 (1.94 and 1.97 in the younger and older cohorts, respectively). Twenty-nine patients (42.6 %; 15 younger and 14 older children, respectively) did not report any bleeding while on N8-GP prophylaxis; 39 patients (57.4 %; 19 younger and 20 older children, respectively) reported 70 bleeds (all mild\/moderate). N8-GP treatment was successful for 78.6 % of bleeds in all patients, 80.0 % in younger and 77.5 % in older patients. Most bleeds (80.0 %) were treated with \u22642 injections. Half-life ratio between N8-GP and the patients\u2019 previous FVIII product was 1.85. N8-GP was well tolerated and provided effective prophylaxis and treatment of bleeds in paediatric patients with severe haemophilia A.<\/jats:p><jats:p>Trial registered at www.clinicaltrials.gov (NCT01731600).<\/jats:p><jats:p>Supplementary Material to this article is available online at www.thrombosis-online.com.<\/jats:p>","DOI":"10.1160\/th17-03-0166","type":"journal-article","created":{"date-parts":[[2017,7,6]],"date-time":"2017-07-06T13:26:05Z","timestamp":1499347565000},"page":"1705-1713","source":"Crossref","is-referenced-by-count":58,"title":["Safety and efficacy of a glycoPEGylated rFVIII (turoctocog alpha pegol, N8-GP) in paediatric patients with severe haemophilia A"],"prefix":"10.1160","volume":"117","author":[{"given":"Sandrine","family":"Meunier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jayanthi","family":"Alamelu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silke","family":"Ehrenforth","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hideji","family":"Hanabusa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Faraizah Abdul","family":"Karim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kaan","family":"Kavakli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melanie","family":"Khodaie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janice","family":"Staber","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Oleksandra","family":"Stasyshyn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Donald","family":"Yee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lina","family":"Rageliene","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"194","published-online":{"date-parts":[[2017,10,26]]},"reference":[{"key":"10.1160\/TH17-03-0166-1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2012.02909.x"},{"key":"10.1160\/TH17-03-0166-2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa067659"},{"key":"10.1160\/TH17-03-0166-3","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12929"},{"key":"10.1160\/TH17-03-0166-4","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(11)61139-2"},{"key":"10.1160\/TH17-03-0166-5","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1182\/asheducation-2011.1.397","volume":"2011","author":"Fogarty","year":"2011","journal-title":"Hematology Am Soc Hematol Educ Program"},{"key":"10.1160\/TH17-03-0166-6","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V98.6.1727"},{"key":"10.1160\/TH17-03-0166-7","doi-asserted-by":"publisher","DOI":"10.1016\/S0049-3848(12)70003-8"},{"key":"10.1160\/TH17-03-0166-8","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2012.02931.x"},{"key":"10.1160\/TH17-03-0166-9","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12405"},{"key":"10.1160\/TH17-03-0166-10","doi-asserted-by":"publisher","DOI":"10.2147\/JBM.S6885"},{"key":"10.1160\/TH17-03-0166-11","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12161"},{"key":"10.1160\/TH17-03-0166-12","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2009.02135.x"},{"key":"10.1160\/TH17-03-0166-13","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-01-407494"},{"key":"10.1160\/TH17-03-0166-14","doi-asserted-by":"publisher","DOI":"10.1160\/TH16-06-0444"},{"key":"10.1160\/TH17-03-0166-15","unstructured":"International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. 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