Primary manuscript

Long-term safety and efficacy of N8-GP in previously treated paediatric patients with haemophilia A: Final results from the pathfinder5 trial

Author
Trakymiene SS, Economou M, Kenet G, Landorph A, Shen C, Kearney S
Citation
Giangrande, P, Abdul Karim, F, Nemes, L, et al. Long-term safety and efficacy of N8-GP in previously treated adults and adolescents with hemophilia A: Final results from pathfinder2. J Thromb Haemost. 2020; 18(Suppl. 1): 5– 14.
Date of Publication
Abstract
Background N8?GP (turoctocog alfa pegol; Esperoct®, Novo Nordisk A/S, Bagsvaerd, Denmark) is a glycoPEGylated, extended half?life human recombinant factor VIII (FVIII). Objective Here, we report end?of?trial safety and efficacy results from the completed N8?GP pathfinder5 trial. Methods pathfinder5 (NCT01731600) was a multi?national, open?label, single?arm, non?randomized, non?controlled trial in previously treated male patients aged <12 years old with severe hemophilia A that comprised a main and an extension phase. During the main phase, patients received twice?weekly N8?GP 60 IU/kg for 50 exposure days (~26 weeks). During the extension phase, patients received the same regimen until the end of trial (first patient in main phase, 20 February 2013; trial end, 28 September 2018). Results Sixty?eight patients were exposed to N8?GP for a median time of ~4.9 years on regimen. Of the 63 patients who started in the extension phase, 62 completed the trial. No FVIII inhibitors (?0.6 BU) or other safety concerns were detected. The overall estimated annualized bleeding rate was 1.08 (median 0.81), and nearly 20% of patients had no bleeds during the entire trial. The proportion of patients with no annual bleeds increased with time, with 56% of patients experiencing no bleeds and 86% experiencing no spontaneous bleeds during the fourth year of exposure. All baseline target joints of patients who participated in both phases of this trial were resolved in slightly over 2 years. Conclusion Overall, data from the completed pathfinder5 trial show that long?term (median 4.9 years) N8?GP treatment was efficacious and well tolerated in previously treated pediatric patients with severe hemophilia A.

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