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A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis
Background rVIII-SingleChain is a recombinant factor VIII (FVIII) with increased binding affinity to von Willebrand factor compared with other FVIII products. rVIII-SingleChain is indicated for the treatment and prevention of bleeding episodes in patients with hemophilia A. Objectives To collect r...
| Autores principales: | , , , , , , , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Georg Thieme Verlag KG
2023
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10113037/ https://www.ncbi.nlm.nih.gov/pubmed/36758611 http://dx.doi.org/10.1055/s-0043-1761449 |
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| author | Guillet, Benoit Hassoun, Abel Wibaut, Bénédicte Harroche, Annie Biron-Andréani, Christine Repesse, Yohan d'Oiron, Roseline Tardy, Brigitte Pan Petesch, Brigitte Chamouni, Pierre Gay, Valérie Fouassier, Marc Pouplard, Claire Martin, Cédric Catovic, Hasan Delavenne, Xavier |
| author_facet | Guillet, Benoit Hassoun, Abel Wibaut, Bénédicte Harroche, Annie Biron-Andréani, Christine Repesse, Yohan d'Oiron, Roseline Tardy, Brigitte Pan Petesch, Brigitte Chamouni, Pierre Gay, Valérie Fouassier, Marc Pouplard, Claire Martin, Cédric Catovic, Hasan Delavenne, Xavier |
| author_sort | Guillet, Benoit |
| collection | PubMed |
| description | Background rVIII-SingleChain is a recombinant factor VIII (FVIII) with increased binding affinity to von Willebrand factor compared with other FVIII products. rVIII-SingleChain is indicated for the treatment and prevention of bleeding episodes in patients with hemophilia A. Objectives To collect real-world evidence data from patients treated with rVIII-SingleChain to confirm the efficacy and safety established in the clinical trial program and carry out a population pharmacokinetic (PK) analysis. Methods This interim analysis includes data, collected between January 2018 — September 2021, from patients treated with rVIII-SingleChain prophylaxis at French Hemophilia Treatment centers. Data on annualized bleeding rates, dosing frequency, and consumption before and after switching to rVIII-SingleChain were recorded. A population PK analysis was also conducted to estimate PK parameters. Results Overall, 43 patients switched to prophylaxis with rVIII-SingleChain either from a previous prophylaxis regimen or from on-demand treatment. Following the switch to rVIII-SingleChain, patients maintained excellent bleed control. After switching to rVIII-SingleChain, most patients maintained or reduced their regimen. Interestingly, a majority of patients treated >2 ×/weekly with a standard half-life FVIII reduced both injection frequency and FVIII consumption with rVIII-SingleChain. A PK analysis revealed a lower clearance of rVIII-SingleChain (1.9 vs. 2.1 dL/h) and a longer half-life both in adolescents/adults ( n = 28) and pediatric ( n = 6) patients (15.5 and 11.9 hours, respectively vs. 14.5 and 10.3 hours) than previously reported. Conclusions Patients who switched to rVIII-SingleChain prophylaxis demonstrated excellent bleed control and a reduction in infusion frequency. A population PK analysis revealed improved PK parameters compared with those reported in the clinical trial. |
| format | Online Article Text |
| id | pubmed-10113037 |
| institution | National Center for Biotechnology Information |
| language | English |
| publishDate | 2023 |
| publisher | Georg Thieme Verlag KG |
| record_format | MEDLINE/PubMed |
| spelling | pubmed-101130372023-04-19 A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis Guillet, Benoit Hassoun, Abel Wibaut, Bénédicte Harroche, Annie Biron-Andréani, Christine Repesse, Yohan d'Oiron, Roseline Tardy, Brigitte Pan Petesch, Brigitte Chamouni, Pierre Gay, Valérie Fouassier, Marc Pouplard, Claire Martin, Cédric Catovic, Hasan Delavenne, Xavier Thromb Haemost Background rVIII-SingleChain is a recombinant factor VIII (FVIII) with increased binding affinity to von Willebrand factor compared with other FVIII products. rVIII-SingleChain is indicated for the treatment and prevention of bleeding episodes in patients with hemophilia A. Objectives To collect real-world evidence data from patients treated with rVIII-SingleChain to confirm the efficacy and safety established in the clinical trial program and carry out a population pharmacokinetic (PK) analysis. Methods This interim analysis includes data, collected between January 2018 — September 2021, from patients treated with rVIII-SingleChain prophylaxis at French Hemophilia Treatment centers. Data on annualized bleeding rates, dosing frequency, and consumption before and after switching to rVIII-SingleChain were recorded. A population PK analysis was also conducted to estimate PK parameters. Results Overall, 43 patients switched to prophylaxis with rVIII-SingleChain either from a previous prophylaxis regimen or from on-demand treatment. Following the switch to rVIII-SingleChain, patients maintained excellent bleed control. After switching to rVIII-SingleChain, most patients maintained or reduced their regimen. Interestingly, a majority of patients treated >2 ×/weekly with a standard half-life FVIII reduced both injection frequency and FVIII consumption with rVIII-SingleChain. A PK analysis revealed a lower clearance of rVIII-SingleChain (1.9 vs. 2.1 dL/h) and a longer half-life both in adolescents/adults ( n = 28) and pediatric ( n = 6) patients (15.5 and 11.9 hours, respectively vs. 14.5 and 10.3 hours) than previously reported. Conclusions Patients who switched to rVIII-SingleChain prophylaxis demonstrated excellent bleed control and a reduction in infusion frequency. A population PK analysis revealed improved PK parameters compared with those reported in the clinical trial. Georg Thieme Verlag KG 2023-02-09 /pmc/articles/PMC10113037/ /pubmed/36758611 http://dx.doi.org/10.1055/s-0043-1761449 Text en The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. ( https://creativecommons.org/licenses/by-nc-nd/4.0/ ) https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License, which permits unrestricted reproduction and distribution, for non-commercial purposes only; and use and reproduction, but not distribution, of adapted material for non-commercial purposes only, provided the original work is properly cited. |
| spellingShingle | Guillet, Benoit Hassoun, Abel Wibaut, Bénédicte Harroche, Annie Biron-Andréani, Christine Repesse, Yohan d'Oiron, Roseline Tardy, Brigitte Pan Petesch, Brigitte Chamouni, Pierre Gay, Valérie Fouassier, Marc Pouplard, Claire Martin, Cédric Catovic, Hasan Delavenne, Xavier A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis |
| title | A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis |
| title_full | A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis |
| title_fullStr | A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis |
| title_full_unstemmed | A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis |
| title_short | A French Real-World Evidence Study Evaluating the Efficacy, Safety, and Pharmacokinetic Parameters of rVIII-SingleChain in Patients with Hemophilia A Receiving Prophylaxis |
| title_sort | french real-world evidence study evaluating the efficacy, safety, and pharmacokinetic parameters of rviii-singlechain in patients with hemophilia a receiving prophylaxis |
| url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10113037/ https://www.ncbi.nlm.nih.gov/pubmed/36758611 http://dx.doi.org/10.1055/s-0043-1761449 |
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