• Nicola Curry, Canan Albayrak, Miguel Escobar, Andre Holme Pål P Department of Haematology, Institute of Clinical Medicine, University of Oslo, Oslo University Hospital, Oslo, Norway., Susan Kearney, Robert Klamroth, Mudi Misgav, Claude Négrier, Allison Wheeler, Elena Santagostino, Midori Shima, Andrea Landorph, Sidsel Marie Tønder, and Steven R Lentz.
• Oxford Haemophilia and Thrombosis Centre and Oxford NIHR BRC, Churchill Hospital, Oxford, UK.
• Haemophilia. 2019 May 1; 25 (3): 373-381.

IntroductionTuroctocog alfa pegol (N8-GP) is a site-specific, 40 kDa glycoPEGylated recombinant factor VIII (FVIII) product with an extended half-life. The comprehensive main phase of the pivotal pathfinder 2 trial showed N8-GP dosed every 4 days (Q4D) provided favourable safety and efficacy for preventing bleeds in 175 patients with haemophilia A.Aim And MethodsWe investigated the safety and efficacy of N8-GP prophylaxis when administered weekly (Q7D) for 24 weeks to patients with low bleeding rates in the pathfinder 2 extension trial. Patients (≥12 years) with ≤2 bleeds during the preceding 6 months of the pathfinder 2 main phase were eligible for randomization to receive N8-GP 50 IU/kg Q4D or 75 IU/kg Q7D. Safety and efficacy endpoints were incidence of FVIII inhibitors and annualized bleeding rate (ABR), respectively.ResultsFifty-five of 143 (38.5%) patients on prophylaxis who continued into the extension phase were randomized to receive 50 IU/kg Q4D (n = 17) or 75 IU/kg Q7D (n = 38). Nine patients in the Q7D cohort reverted to 50 IU/kg Q4D. No inhibitors were detected. In both cohorts, >50% of patients experienced no bleeds. Median ABR for overall, joint, spontaneous, traumatic and muscle was 0.00 for both cohorts. Overall estimated success rate for treating bleeding episodes was 87.5%; 94.7% of bleeds were controlled with ≤2 injections.ConclusionsWeekly N8-GP was well tolerated and efficacious and may benefit selected "low bleeder" patients with haemophilia A.© 2019 The Authors. Haemophilia Published by John Wiley & Sons Ltd.

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