An evidence based therapeutic approach to hereditary and acquired angioedema.

Abstract

Hereditary angioedema (HAE) due to C1 esterase inhibitor (C1-INH) deficiency (HAE-C1-INH), HAE with normal C1-INH, and acquired angioedema due to C1-INH deficiency are rare but important diseases that can be associated with significant morbidity and mortality. Research into the pathogenesis of angioedema has expanded greatly and has led to new clinical trials with novel therapeutic agents and strategies. Strategies for managing HAE-C1-INH are aimed at treating acute attacks or preventing attacks through the use of prophylactic treatment. Agents available in Europe for treating acute attacks include plasma-derived C1-INH concentrates, a bradykinin B2 receptor (B2R) antagonist, and a recombinant human C1-INH. In the USA, a plasma-derived C1-INH concentrate, a bradykinin B2R antagonist, and a plasma kallikrein inhibitor have been approved for the treatment of acute HAE-C1-INH attacks. C1-INH concentrates and attenuated androgens are used for short-term prophylactic treatment. Long-term prophylactic treatments include attenuated androgens, a plasma-derived C1-INH concentrate, and antifibrinolytics. Plasma-derived C1-INH and a bradykinin B2R antagonist are approved for self-administration at home. The number of management options for HAE-C1-INH and similar conditions has increased considerably within the last few years, thus helping to alleviate the burden of these rare diseases.