Immunogenicity of Current and New Therapies for Hemophilia A
Anti-drug antibody (ADA) development is a significant complication in the treatment of several conditions. For decades, the mainstay of hemophilia A treatment was the replacement of deficient coagulation factor VIII (FVIII) to restore hemostasis, control, and prevent bleeding events. Recently, new p...
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| Format: | Article |
| Language: | English |
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MDPI AG
2022-07-01
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| Series: | Pharmaceuticals |
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| Online Access: | https://www.mdpi.com/1424-8247/15/8/911 |
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| author | Alessandra N. L. Prezotti Jéssica O. Frade-Guanaes Gabriela G. Yamaguti-Hayakawa Margareth C. Ozelo |
| author_facet | Alessandra N. L. Prezotti Jéssica O. Frade-Guanaes Gabriela G. Yamaguti-Hayakawa Margareth C. Ozelo |
| author_sort | Alessandra N. L. Prezotti |
| collection | DOAJ |
| description | Anti-drug antibody (ADA) development is a significant complication in the treatment of several conditions. For decades, the mainstay of hemophilia A treatment was the replacement of deficient coagulation factor VIII (FVIII) to restore hemostasis, control, and prevent bleeding events. Recently, new products have emerged for hemophilia A replacement therapy, including bioengineered FVIII molecules with enhanced pharmacokinetic profiles: the extended half-life (EHL) recombinant FVIII products. However, the main complication resulting from replacement treatment in hemophilia A is the development of anti-FVIII neutralizing alloantibodies, known as inhibitors, affecting approximately 25–30% of severe hemophilia A patients. Therefore, the immunogenicity of each FVIII product and the mechanisms that could help increase the tolerance to these products have become important research topics in hemophilia A. Furthermore, patients with inhibitors continue to require effective treatment for breakthrough bleedings and procedures, despite the availability of non-replacement therapy, such as emicizumab. Herein, we discuss the currently licensed treatments available for hemophilia A and the immunogenicity of new therapies, such as EHL-rFVIII products, compared to other products available. |
| first_indexed | 2024-03-09T09:50:42Z |
| format | Article |
| id | doaj.art-2d71683a1efa44009d4be958ca6101ba |
| institution | Directory Open Access Journal |
| issn | 1424-8247 |
| language | English |
| last_indexed | 2024-03-09T09:50:42Z |
| publishDate | 2022-07-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceuticals |
| spelling | doaj.art-2d71683a1efa44009d4be958ca6101ba2023-12-02T00:08:36ZengMDPI AGPharmaceuticals1424-82472022-07-0115891110.3390/ph15080911Immunogenicity of Current and New Therapies for Hemophilia AAlessandra N. L. Prezotti0Jéssica O. Frade-Guanaes1Gabriela G. Yamaguti-Hayakawa2Margareth C. Ozelo3Department of Internal Medicine, School of Medical Sciences, University of Campinas, UNICAMP, Rua Tessália Vieira de Camargo, 126, Cidade Universitária, Campinas 13083-887, SP, BrazilDepartment of Internal Medicine, School of Medical Sciences, University of Campinas, UNICAMP, Rua Tessália Vieira de Camargo, 126, Cidade Universitária, Campinas 13083-887, SP, BrazilDepartment of Internal Medicine, School of Medical Sciences, University of Campinas, UNICAMP, Rua Tessália Vieira de Camargo, 126, Cidade Universitária, Campinas 13083-887, SP, BrazilDepartment of Internal Medicine, School of Medical Sciences, University of Campinas, UNICAMP, Rua Tessália Vieira de Camargo, 126, Cidade Universitária, Campinas 13083-887, SP, BrazilAnti-drug antibody (ADA) development is a significant complication in the treatment of several conditions. For decades, the mainstay of hemophilia A treatment was the replacement of deficient coagulation factor VIII (FVIII) to restore hemostasis, control, and prevent bleeding events. Recently, new products have emerged for hemophilia A replacement therapy, including bioengineered FVIII molecules with enhanced pharmacokinetic profiles: the extended half-life (EHL) recombinant FVIII products. However, the main complication resulting from replacement treatment in hemophilia A is the development of anti-FVIII neutralizing alloantibodies, known as inhibitors, affecting approximately 25–30% of severe hemophilia A patients. Therefore, the immunogenicity of each FVIII product and the mechanisms that could help increase the tolerance to these products have become important research topics in hemophilia A. Furthermore, patients with inhibitors continue to require effective treatment for breakthrough bleedings and procedures, despite the availability of non-replacement therapy, such as emicizumab. Herein, we discuss the currently licensed treatments available for hemophilia A and the immunogenicity of new therapies, such as EHL-rFVIII products, compared to other products available.https://www.mdpi.com/1424-8247/15/8/911hemophiliafactor VIIIblood coagulation factorsinhibitorsimmunogenicityplasma-derived factor VIII |
| spellingShingle | Alessandra N. L. Prezotti Jéssica O. Frade-Guanaes Gabriela G. Yamaguti-Hayakawa Margareth C. Ozelo Immunogenicity of Current and New Therapies for Hemophilia A Pharmaceuticals hemophilia factor VIII blood coagulation factors inhibitors immunogenicity plasma-derived factor VIII |
| title | Immunogenicity of Current and New Therapies for Hemophilia A |
| title_full | Immunogenicity of Current and New Therapies for Hemophilia A |
| title_fullStr | Immunogenicity of Current and New Therapies for Hemophilia A |
| title_full_unstemmed | Immunogenicity of Current and New Therapies for Hemophilia A |
| title_short | Immunogenicity of Current and New Therapies for Hemophilia A |
| title_sort | immunogenicity of current and new therapies for hemophilia a |
| topic | hemophilia factor VIII blood coagulation factors inhibitors immunogenicity plasma-derived factor VIII |
| url | https://www.mdpi.com/1424-8247/15/8/911 |
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