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Update on recent trials in the treatment of hereditary angioedema

Neelu Kalra,Timothy Craig

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 lowplasma 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 options expand, selection of therapy for both prophylaxis and for acute attacks will require optimization based on patient- and drug-specific factors. In this article we provide an overview of the latest developments on therapeutic options and emerging trends in overall management of HAE.