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Human Plasma-Derived, Nanofiltered, C1-Inhibitor Concentrate (Cinryze®), a Novel Therapeutic Alternative for the Management of Hereditary Angioedema Resulting from C1-Inhibitor Deficiency
Hereditary angioedema resulting from the deficiency of the C1 inhibitor (HAE-C1-INH) is a rare, but potentially life-threatening disorder characterized by paroxysmal episodes of subcutaneous or submucosal edema. Early diagnosis is essential. Management is aimed at the prompt elimination of full-fled...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Springer Healthcare Communications
2012
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3906706/ https://www.ncbi.nlm.nih.gov/pubmed/24490128 http://dx.doi.org/10.1007/s13554-012-0002-5 |
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author | Farkas, Henriette Varga, Lilian |
author_facet | Farkas, Henriette Varga, Lilian |
author_sort | Farkas, Henriette |
collection | PubMed |
description | Hereditary angioedema resulting from the deficiency of the C1 inhibitor (HAE-C1-INH) is a rare, but potentially life-threatening disorder characterized by paroxysmal episodes of subcutaneous or submucosal edema. Early diagnosis is essential. Management is aimed at the prompt elimination of full-fledged attacks, as well as at the prevention of edematous episodes. The most straightforward means for therapy is supplementation with the deficient C1-INH protein. Placebo-controlled and open clinical studies have established that nanofiltered, human C1-INH concentrate, Cinryze® (ViroPharma Inc., Exton, PA, USA) (C1-INH(Ci)), administered in 1,000 U doses is an effective and safe remedy for edematous episodes of HAE-C1-INH, regardless of the localization of the attack. Clinical manifestations rapidly improve and then resolve completely following treatment with this medicinal product. Additionally, C1-INH(Ci) is also appropriate for pre-procedural or for routine prophylaxis. The administration of 1,000 U C1-INH(Ci) before the (dental, surgical, or interventional diagnostic) procedure reduced the incidence of edematous episodes compared with placebo, and this reduction proved significant during routine prophylaxis with the administration of this dose every 3–4 days. Relapses did not occur, and repeated dosing had no influence on the efficacy of the preparation. Patients also tolerated treatment with C1-INH(Ci) well. The safety of this preparation was confirmed by the absence of viral transmission as well as by the lack of antibody formation against C1-INH during treatment. Nowadays, C1-INH(Ci) for intravenous use is the only medicinal product indicated both for the prevention and management of edematous attacks. |
format | Online Article Text |
id | pubmed-3906706 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2012 |
publisher | Springer Healthcare Communications |
record_format | MEDLINE/PubMed |
spelling | pubmed-39067062014-01-30 Human Plasma-Derived, Nanofiltered, C1-Inhibitor Concentrate (Cinryze®), a Novel Therapeutic Alternative for the Management of Hereditary Angioedema Resulting from C1-Inhibitor Deficiency Farkas, Henriette Varga, Lilian Biol Ther Review Hereditary angioedema resulting from the deficiency of the C1 inhibitor (HAE-C1-INH) is a rare, but potentially life-threatening disorder characterized by paroxysmal episodes of subcutaneous or submucosal edema. Early diagnosis is essential. Management is aimed at the prompt elimination of full-fledged attacks, as well as at the prevention of edematous episodes. The most straightforward means for therapy is supplementation with the deficient C1-INH protein. Placebo-controlled and open clinical studies have established that nanofiltered, human C1-INH concentrate, Cinryze® (ViroPharma Inc., Exton, PA, USA) (C1-INH(Ci)), administered in 1,000 U doses is an effective and safe remedy for edematous episodes of HAE-C1-INH, regardless of the localization of the attack. Clinical manifestations rapidly improve and then resolve completely following treatment with this medicinal product. Additionally, C1-INH(Ci) is also appropriate for pre-procedural or for routine prophylaxis. The administration of 1,000 U C1-INH(Ci) before the (dental, surgical, or interventional diagnostic) procedure reduced the incidence of edematous episodes compared with placebo, and this reduction proved significant during routine prophylaxis with the administration of this dose every 3–4 days. Relapses did not occur, and repeated dosing had no influence on the efficacy of the preparation. Patients also tolerated treatment with C1-INH(Ci) well. The safety of this preparation was confirmed by the absence of viral transmission as well as by the lack of antibody formation against C1-INH during treatment. Nowadays, C1-INH(Ci) for intravenous use is the only medicinal product indicated both for the prevention and management of edematous attacks. Springer Healthcare Communications 2012-05-09 /pmc/articles/PMC3906706/ /pubmed/24490128 http://dx.doi.org/10.1007/s13554-012-0002-5 Text en © The Author(s) 2012 https://creativecommons.org/licenses/by-nc/4.0/ Open Access. This article is distributed under the terms of the Creative Commons Attribution Noncommercial License which permits any noncommercial use, distribution, and reproduction in any medium, provided the original author(s) and source are credited. |
spellingShingle | Review Farkas, Henriette Varga, Lilian Human Plasma-Derived, Nanofiltered, C1-Inhibitor Concentrate (Cinryze®), a Novel Therapeutic Alternative for the Management of Hereditary Angioedema Resulting from C1-Inhibitor Deficiency |
title | Human Plasma-Derived, Nanofiltered, C1-Inhibitor Concentrate (Cinryze®), a Novel Therapeutic Alternative for the Management of Hereditary Angioedema Resulting from C1-Inhibitor Deficiency |
title_full | Human Plasma-Derived, Nanofiltered, C1-Inhibitor Concentrate (Cinryze®), a Novel Therapeutic Alternative for the Management of Hereditary Angioedema Resulting from C1-Inhibitor Deficiency |
title_fullStr | Human Plasma-Derived, Nanofiltered, C1-Inhibitor Concentrate (Cinryze®), a Novel Therapeutic Alternative for the Management of Hereditary Angioedema Resulting from C1-Inhibitor Deficiency |
title_full_unstemmed | Human Plasma-Derived, Nanofiltered, C1-Inhibitor Concentrate (Cinryze®), a Novel Therapeutic Alternative for the Management of Hereditary Angioedema Resulting from C1-Inhibitor Deficiency |
title_short | Human Plasma-Derived, Nanofiltered, C1-Inhibitor Concentrate (Cinryze®), a Novel Therapeutic Alternative for the Management of Hereditary Angioedema Resulting from C1-Inhibitor Deficiency |
title_sort | human plasma-derived, nanofiltered, c1-inhibitor concentrate (cinryze®), a novel therapeutic alternative for the management of hereditary angioedema resulting from c1-inhibitor deficiency |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3906706/ https://www.ncbi.nlm.nih.gov/pubmed/24490128 http://dx.doi.org/10.1007/s13554-012-0002-5 |
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