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01.12.2008 | Leading Article

Current and Emerging Management Options for Hereditary Angioedema in the US

verfasst von: Tolly G. Epstein, Dr Jonathan A. Bernstein

Erschienen in: Drugs | Ausgabe 18/2008

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Abstract

Hereditary angioedema (HAE) is a rare disorder characterized by recurrent attacks of swelling that may involve multiple anatomical locations. In the majority of patients, it is caused by a functional or quantitative defect in the C1 inhibitor (C1-INH), which is an important regulator of the complement, fibrinolytic, kallikrein-kinin and coagulation systems. Standard treatments used for other types of angioedema are ineffective for HAE. Traditional therapies for HAE, including fresh frozen plasma, ε-aminocaproic acid and danazol, may be well tolerated and effective in some patients; however, there are limitations both in their safety and efficacy.

Several novel therapies have completed phase III trials in the US, including: (i) plasma-derived C1-INH replacement therapies (Berinert P® and Cinryze®); (ii) a recombinant C1-INH replacement therapy (conestat alfa; Rhucin®); (iii) a kallikrein inhibitor (ecallantide [DX-88]); and (iv) a bradykinin-2-receptor antagonist (icatibant). Both Berinert P® and Cinryze® are reported to have excellent efficacy and safety data from phase III trials. Currently, only Cinryze® has been approved for prophylactic use in the US. US FDA approval for other novel agents to treat HAE and for the use of Cinryze® in the treatment of acute attacks is pending.

The use of trade names is for identification purposes only and does not imply endorsement.

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Titel: Current and Emerging Management Options for Hereditary Angioedema in the US
verfasst von: Tolly G. Epstein
Dr Jonathan A. Bernstein
Publikationsdatum: 01.12.2008
Verlag: Springer International Publishing
Erschienen in: Drugs / Ausgabe 18/2008
Print ISSN: 0012-6667
Elektronische ISSN: 1179-1950
DOI: https://doi.org/10.2165/0003495-200868180-00003

Springer Medizin

Abstract

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