• Sandrine Meunier, Jayanthi Alamelu, Silke Ehrenforth, Hideji Hanabusa, Faraizah Abdul Karim, Kaan Kavakli, Melanie Khodaie, Janice Staber, Oleksandra Stasyshyn, Donald L Yee, and Lina Rageliene.
• Dr Sandrine Meunier, Unité d'Hémostase Clinique, Centre Régional de Traitement de l'Hémophilie, Hospices Civils de Lyon, Groupement Hospitalier Est, Hôpital Louis PRADEL, 59 Boulevard Pinel, 69677 BRON Cedex, France, Tel.: +33 472 118810, Fax: +33 472 118817, E-mail: sandrine.meunier@chu-lyon.fr.
• Thromb. Haemost. 2017 Aug 30; 117 (9): 1705-1713.

AbstractTuroctocog alfa pegol (N8-GP, Novo Nordisk, Bagsværd, Denmark), an extended half-life glycoPEGylated recombinant factor VIII (rFVIII), is being developed for prophylaxis and treatment of bleeds in haemophilia A patients. pathfinder™5 is a multinational, open-label, single-arm trial to assess safety, efficacy and pharmacokinetics of N8-GP in paediatric (<12 years), previously treated patients. Boys with severe haemophilia A (<1 % FVIII), no history of inhibitors and previously treated with FVIII products (>50 exposure days [ED] for patients aged 0-5 years [younger cohort]; >150 ED for patients aged 6-11 years [older cohort]) were included. For prophylaxis, N8-GP was dosed at 50-75 IU/kg twice weekly; bleeds were treated with 20-75 IU/kg. Half-life was estimated for the patients' 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 ≤2 injections. Half-life ratio between N8-GP and the patients' 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.

34 keywords...

hide…