Case Reports
Vol. 3 No. 1 (2024)
Tailored therapy with turoctocog alfa pegol according to patient’s lifestyle and hemorrhagic phenotype: from clinical trial to real-life
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All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.
All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.
Received: 14 December 2023
Accepted: 18 March 2024
Accepted: 18 March 2024
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Although the use of prophylaxis regimens with prolonged half-life factors is now widespread in the world of hemophilia A, there is a lack of real-life evidence on the impact of these products on joint health, adherence, and quality of life of patients. Turoctocog alfa pegol is a glycoPEGylated recombinant factor VIII (FVIII) with an extended half-life (EHL), developed for prophylaxis, treatment of bleeds, and perioperative management in patients with hemophilia A. We report here on three cases of three patients affected by severe hemophilia A, with variable bleeding phenotype and lifestyle, to describe our clinical practice on prophylaxis with turoctocog alfa pegol. As confirmed in our cases, FVIII trough levels remained coherent with those experienced in the registration trial after the switch to the commercial EHL drug. Moreover, the cases highlight how the current clinical management of hemophilia can personalize treatment in several specific conditions.
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Citations
Giangrande P, Andreeva T, Chowdary P, et al. Clinical evaluation of glycoPEGylated recombinant FVIII: efficacy and safety in severe haemophilia A. Thromb Haemost 2017;117:252-61. DOI: https://doi.org/10.1160/TH16-06-0444
Tiede A, Brand B, Fischer R, et al. Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A. J Thromb Haemost 2013;11:670-8. DOI: https://doi.org/10.1111/jth.12161
Tosetto A, Neff A, Lentz SR, et al. Turoctocog alfa pegol provides effective management for major and minor surgical procedures in patients across all age groups with severe haemophilia A: Full data set from the pathfinder 3 and 5 phase III trials. Haemophilia 2020;26:450-8. DOI: https://doi.org/10.1111/hae.13980
U.S. Food and Drug Administration. Esperoct prescribing information. Available from: https://www.fda.gov/media/120351/download (accessed on October 13th, 2021)
European Medicines Agency. Esperoct. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/esperoct (accessed on October 13th, 2021)
Meunier S, Alamelu J, Ehrenforth S, et al. Safety and efficacy of a glycoPEGylated rFVIII (turoctocog alpha pegol, N8-GP) in paediatric patients with severe haemophilia A. Thromb Haemost 2017;117:1705-13. DOI: https://doi.org/10.1160/TH17-03-0166
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;18:5-14. DOI: https://doi.org/10.1111/jth.14959
Iorio A, Keepanasseril A, Foster G, et al. Development of a web-accessible population pharmacokinetic service-hemophilia (WAPPS-Hemo): study protocol. JMIR Res Protoc 2016;e239. DOI: https://doi.org/10.2196/resprot.6558
How to Cite
1.
Nichele I, Carli G, Tosetto A. Tailored therapy with turoctocog alfa pegol according to patient’s lifestyle and hemorrhagic phenotype: from clinical trial to real-life. Bleeding Thromb Vascul Biol [Internet]. 2024 Apr. 22 [cited 2025 Sep. 6];3(1). Available from: https://www.btvb.org/btvb/article/view/104
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