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Published in: Drugs 18/2008

01-12-2008 | Leading Article

Current and Emerging Management Options for Hereditary Angioedema in the US

Authors: Tolly G. Epstein, Dr Jonathan A. Bernstein

Published in: Drugs | Issue 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.
Footnotes
1
The use of trade names is for identification purposes only and does not imply endorsement.
 
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Metadata
Title
Current and Emerging Management Options for Hereditary Angioedema in the US
Authors
Tolly G. Epstein
Dr Jonathan A. Bernstein
Publication date
01-12-2008
Publisher
Springer International Publishing
Published in
Drugs / Issue 18/2008
Print ISSN: 0012-6667
Electronic ISSN: 1179-1950
DOI
https://doi.org/10.2165/0003495-200868180-00003

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