https://doi.org/10.4081/btvb.2024.104
Tailored therapy with turoctocog alfa pegol according to patient’s lifestyle and hemorrhagic phenotype: from clinical trial to real-life
Submitted: 14 December 2023
Accepted: 18 March 2024
Published: 22 April 2024
Accepted: 18 March 2024
Abstract Views: 365
PDF: 107
Publisher's note
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.
Authors
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.
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
Nichele, I., Carli, G., & Tosetto, A. (2024). Tailored therapy with turoctocog alfa pegol according to patient’s lifestyle and hemorrhagic phenotype: from clinical trial to real-life. Bleeding, Thrombosis and Vascular Biology, 3(1). https://doi.org/10.4081/btvb.2024.104
Copyright (c) 2024 The Author(s)
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
PAGEPress has chosen to apply the Creative Commons Attribution NonCommercial 4.0 International License (CC BY-NC 4.0) to all manuscripts to be published.
Most read articles by the same author(s)
- Cristina Legnani, Michela Cini, Sophie Testa, Alberto Tosetto, Claudia Dellanoce, Stefania Bellesso, Giuseppe Carli, Ilaria Nichele, Laura Lissandrini, Serena Zorzi, Emilia Antonucci, Gualtiero Palareti, Evaluation of Coaguchek®Pro II coagulation testing device performance to assess direct oral anticoagulant action. The DOAC-CHECK study , Bleeding, Thrombosis and Vascular Biology: Vol. 1 No. 3 (2022)
- Gualtiero Palareti, Paolo Prandoni, Cristina Legnani, Emilia Antonucci, Serena Zorzi, Alberto Tosetto, Lorenza Bertù, Sophie Testa, Vittorio Pengo, Walter Ageno, Ida Martinelli, Benilde Cosmi, Eugenio Bucherini, Daniela Poli, Rationale and design of a study on D-dimer use to stratify patients after a first unprovoked venous thromboembolism for their risk of recurrence: extended low-dose Apixaban given only to patients with positive D-dimer results , Bleeding, Thrombosis and Vascular Biology: Vol. 1 No. 1 (2022)
Similar Articles
- Giovanni de Gaetano, Well begun is half done , Bleeding, Thrombosis and Vascular Biology: Vol. 1 No. 3 (2022)
- Marialaura Bonaccio, Licia Iacoviello, Maria Benedetta Donati, It’s definitely time to consider diet in its ultra-processing form as a major risk factor for thrombotic vascular disorders , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 3 (2023)
- Massimo Franchini, Polycythemia vera and management of the thrombotic risk: an update , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 4 (2023)
- Paolo Gresele, Artificial intelligence and machine learning in hemostasis and thrombosis , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 4 (2023)
- Ang Li, Emily Zhou, Trends and updates on the epidemiology of cancer-associated thrombosis: a systematic review , Bleeding, Thrombosis and Vascular Biology: Vol. 3 No. s1 (2024): 12th ICTHIC
- Nadim Tawil, Lata Adnani, Janusz Rak, Coagulome and tumor microenvironment: impact of oncogenes, cellular heterogeneity and extracellular vesicles , Bleeding, Thrombosis and Vascular Biology: Vol. 3 No. s1 (2024): 12th ICTHIC
- Anna Falanga, Hugo Ten Cate, Bianca Rocca, How to manage antithrombotic treatments in thrombocytopenic patients with cancer. Comments on the European Haematology Association guidelines , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 1 (2023)
- Maria Barca-Hernando, Victor Garcia-Garcia, Luis Jara-Palomares, Location of metastasis and complications in patients with venous thromboembolism and cancer: a systematic review , Bleeding, Thrombosis and Vascular Biology: Vol. 3 No. s1 (2024): 12th ICTHIC
- Daniela Poli, Riccardo Tartaglia, Doris Barcellona , Paolo Bucciarelli , Antonio Ciampa, Elvira Grandone, Giuseppe Malcangi, Giuseppe Rescigno, Vincenzo Toschi, Sophie Testa, Alessandro Squizzato, Attitude to clinical research among health professionals affiliated with the Italian Federation of Centers for the Diagnosis of Thrombotic Disorders and the Surveillance of the Antithrombotic Therapies (FCSA) , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 1 (2023)
- Gualtiero Palareti, The diagnostic procedure for subjects with suspected pulmonary embolism. A recent comparison among the recommendations available from the international guidelines , Bleeding, Thrombosis and Vascular Biology: Vol. 3 No. 1 (2024)
You may also start an advanced similarity search for this article.
