The New Era of C1-Esterase Inhibitor Deficiency Therapy

Abstract

Hereditary angioedema (HAE) is an autosomal dominant disorder clinically characterized by recurrent episodes of angioedema. Until late-2008, HAE therapy in the United States was largely limited to antifibrinolytic agents or attenuated androgens. Although these drugs decrease the number and severity of angioedema attacks, they are associated with significant dose-related adverse effects. Recent advances have dramatically changed the management of HAE. As a result, we are embarking on a new era of treatment for this condition that includes effective on-demand treatment of attacks as well as effective prophylactic treatment. Herein we discuss the various treatment options for C1-inhibitor deficiency, focusing on new developments and literature published over the past year, as well as the additional patient considerations that should be addressed when determining the most appropriate patient-specific treatment plan.