1. Safety and efficacy of a glycoPEGylated rFVIII (turoctocog alpha pegol, N8-GP) in paediatric patients with severe haemophilia A
- Author
-
Sandrine Meunier, Lina Rageliene, Kaan Kavakli, Donald L. Yee, Jayanthi Alamelu, Oleksandra Stasyshyn, Melanie Khodaie, Silke Ehrenforth, Hideji Hanabusa, Janice M. Staber, and Faraizah Abdul Karim
- Subjects
Male ,Pediatrics ,medicine.medical_specialty ,Asia ,Haemophilia A ,Alpha (ethology) ,030204 cardiovascular system & hematology ,Hemophilia A ,Haemophilia ,Risk Assessment ,Severity of Illness Index ,03 medical and health sciences ,0302 clinical medicine ,Pharmacokinetics ,Hemarthrosis ,medicine ,Humans ,Child ,Paediatric patients ,Factor VIII ,Coagulants ,business.industry ,Infant, Newborn ,Infant ,Hematology ,Turoctocog alfa ,medicine.disease ,Europe ,Treatment Outcome ,Child, Preschool ,North America ,Cohort ,Severe haemophilia A ,Patient Safety ,business ,Half-Life ,030215 immunology - Abstract
SummaryTuroctocog 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 (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.Trial registered at www.clinicaltrials.gov (NCT01731600).Supplementary Material to this article is available online at www.thrombosis-online.com.
- Published
- 2017