Pasteurized C1 inhibitor concentrate in hereditary angioedema: pharmacology, safety, efficacy and future directions.
Hereditary angioedema (HAE) is a relatively rare genetic disorder that is most commonly caused by a deficiency of C1 inhibitor. It is estimated that HAE affects at least one in 10,000 to one in 50,000 of the worldwide population, with relapsing swelling of the skin and abdominal pain attacks being the most common clinical symptoms. Most seriously, laryngeal edema associated with HAE may lead to death. Replacement therapy with intravenous pasteurized C1 inhibitor concentrate is the recommended treatment for acute attacks of HAE, resulting in a rapid resolution of symptoms. Pasteurized C1 inhibitor concentrates can also be used for prophylaxis of HAE, and are currently also being assessed for home therapy in this setting. Future advances may improve disease burden and mortality associated with HAE.
Bork
Konrad
K
Department of Dermatology, Johannes Gutenberg University, Langenbeckstr. 1, 55131 Mainz, Germany. bork@hautklinik.klinik.uni-mainz.de