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Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety
Hereditary angioedema (HAE) is a clinical disorder characterized by a deficiency of C1 esterase inhibitor (C1-INH). HAE has traditionally been divided into two subtypes. Unique among the inherited deficiencies of the complement system, HAE Types I and II are inherited as an autosomal dominant disord...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Dove Medical Press
2010
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3262319/ https://www.ncbi.nlm.nih.gov/pubmed/22282695 http://dx.doi.org/10.2147/JBM.S9576 |
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author | Lunn, Michael Santos, Carah Craig, Timothy |
author_facet | Lunn, Michael Santos, Carah Craig, Timothy |
author_sort | Lunn, Michael |
collection | PubMed |
description | Hereditary angioedema (HAE) is a clinical disorder characterized by a deficiency of C1 esterase inhibitor (C1-INH). HAE has traditionally been divided into two subtypes. Unique among the inherited deficiencies of the complement system, HAE Types I and II are inherited as an autosomal dominant disorder. The generation of an HAE attack is caused by the depletion and/or consumption of C1-inhibitor manifested as subcutaneous or submucosal edema of the upper airway, face, extremities, or gastrointestinal tract. Attacks can be severe and potentially life-threatening, particularly with laryngeal involvement. Despite the availability of C1-INH for the treatment of HAE since the 1980s in Europe and other countries, HAE treatment in the United States was limited to androgen therapy. The human plasma-derived C1 esterase inhibitor (Cinryze™), distributed by Lev Pharmaceuticals, was approved in October 2008 for the prevention of HAE attacks based on the results of a phase III clinical trial. This review aims to describe the history of C1-INH replacement in HAE as well as the pharmacology, efficacy and safety of C1-INH, concentrating on Cinryze as the first approved chronic replacement treatment for the prophylaxis of HAE attacks. |
format | Online Article Text |
id | pubmed-3262319 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2010 |
publisher | Dove Medical Press |
record_format | MEDLINE/PubMed |
spelling | pubmed-32623192012-01-26 Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety Lunn, Michael Santos, Carah Craig, Timothy J Blood Med Review Hereditary angioedema (HAE) is a clinical disorder characterized by a deficiency of C1 esterase inhibitor (C1-INH). HAE has traditionally been divided into two subtypes. Unique among the inherited deficiencies of the complement system, HAE Types I and II are inherited as an autosomal dominant disorder. The generation of an HAE attack is caused by the depletion and/or consumption of C1-inhibitor manifested as subcutaneous or submucosal edema of the upper airway, face, extremities, or gastrointestinal tract. Attacks can be severe and potentially life-threatening, particularly with laryngeal involvement. Despite the availability of C1-INH for the treatment of HAE since the 1980s in Europe and other countries, HAE treatment in the United States was limited to androgen therapy. The human plasma-derived C1 esterase inhibitor (Cinryze™), distributed by Lev Pharmaceuticals, was approved in October 2008 for the prevention of HAE attacks based on the results of a phase III clinical trial. This review aims to describe the history of C1-INH replacement in HAE as well as the pharmacology, efficacy and safety of C1-INH, concentrating on Cinryze as the first approved chronic replacement treatment for the prophylaxis of HAE attacks. Dove Medical Press 2010-08-24 /pmc/articles/PMC3262319/ /pubmed/22282695 http://dx.doi.org/10.2147/JBM.S9576 Text en © 2010 Lunn et al, publisher and licensee Dove Medical Press Ltd. This is an Open Access article which permits unrestricted noncommercial use, provided the original work is properly cited. |
spellingShingle | Review Lunn, Michael Santos, Carah Craig, Timothy Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety |
title | Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety |
title_full | Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety |
title_fullStr | Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety |
title_full_unstemmed | Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety |
title_short | Cinryze(™) as the first approved C1 inhibitor in the USA for the treatment of hereditary angioedema: approval, efficacy and safety |
title_sort | cinryze(™) as the first approved c1 inhibitor in the usa for the treatment of hereditary angioedema: approval, efficacy and safety |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3262319/ https://www.ncbi.nlm.nih.gov/pubmed/22282695 http://dx.doi.org/10.2147/JBM.S9576 |
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