Hereditary angioedema: on-demand treatment and long-term prophylaxis-a global reality.

Abstract

Hereditary angioedema (HAE) is a rare disorder of recurrent swellings of the subcutaneous and/or mucosal tissues due to defects in the contact system regulation of bradykinin production. The swellings can be disfiguring, cause debilitating abdominal pain, or be fatal with laryngeal obstruction. Over the last decade, there have been immense successes in management of HAE owing to the development of drugs targeting the contact system. This review focuses on management of HAE due to C1-esterase inhibitor (C1-INH) deficiency/dysfunction, including a global perspective which is under-represented in the literature. HAE with normal C1-INH is outside the scope of this article. The review provides summaries of clinical trials that have led to licensing of new HAE medications and discusses access to these modern drugs from a global perspective.