Update on recent trials in the treatment of hereditary angioedema

Abstract

Hereditary angioedema (HAE) is a rare genetic condition that manifests as painful and potentially life-threatening episodic attacks of cutaneous and submucosal swelling. It results from functional deficiency of C1 esterase inhibitor (C1-INH), which is a regulator of the complement system, contact/kinin system and coagulation system. In HAE patients, the low-plasma concentration of functional C1-INH leads to overactivation of the kinin cascade and local release of bradykinin. Bradykinin is responsible for the pain, vascular permeability changes and edema noted in the disease. Until recently, therapeutic options for HAE have been very limited. Many new therapies such as C1-INH replacement drugs and medications aimed at components of the contact system such as plasma kallikrein inhibitors and bradykinin BR2-receptor antagonists, have emerged and will be the focus of this manuscript. We believe availability of new, safe and effective treatment options will change the treatment paradigm of HAE. As therapeutic ...