Title Long-term safety and efficacy of N8-GP in previously treated pediatric patients with hemophilia A: Final results from pathfinder5 /
Authors Šaulytė Trakymienė, Sonata ; Economou, Marina ; Kenet, Gili ; Landorph, Andrea ; Shen, Chunduo ; Kearney, Susan
DOI 10.1111/jth.15036
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Is Part of Journal of thrombosis and haemostasis: Special Issue: N8‐GP (turoctocog alfa pegol; Esperoct®): A state‐of‐the‐art extended half‐life recombinant factor VIII molecule for patients with hemophilia A.. Hoboken : Wiley. 2020, vol. 18, suppl. 1, p. 15-25.. ISSN 1538-7933. eISSN 1538-7836
Keywords [eng] child ; clinical trial ; factor VIII ; hemophilia A ; turoctocog alfa pegol
Abstract [eng] 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.
Published Hoboken : Wiley
Type Journal article
Language English
Publication date 2020
CC license CC license description