PHARMACOTHERAPY AND EVIDENCE BASED MEDICINE: Edited by David A. Khan and Enrico CompalatiDiagnostic and therapeutic management of hereditary angioedema due to C1-inhibitor deficiency the Italian experienceCancian, Mauro and the Italian network for C1-INH-HAE (ITACA) Author Information Dipartimento di Medicina, Università degli Studi di Padova, Padova, Italy Correspondence to Mauro Cancian, Dipartimento di Medicina, Università degli Studi di Padova, Via Giustiniani 2, I-35128 Padova, Italy. Tel: +39 049 8214452; e-mail: [email protected] Current Opinion in Allergy and Clinical Immunology 15(4):p 383-391, August 2015. | DOI: 10.1097/ACI.0000000000000186 Buy Metrics Abstract Purpose of review Hereditary angioedema (HAE) due to C1-inhibitor (C1-INH) deficiency (C1-INH-HAE) is a rare disease, with a reported prevalence of about 1 : 50 000. C1-INH-HAE causes disabling symptoms, which may be life-threatening if swelling affects upper airways. Diagnostic procedures are now well established and the role of bradykinin as the main mediator of plasma outflow eliciting angioedema formation has been clearly elucidated. Recent findings Increased understanding of the pathogenesis of C1-INH-HAE allowed in recent years the development of new drugs targeted to inhibit bradykinin synthesis (Ecallantide) or activity (Icatibant). At the same time, a recombinant C1-INH concentrate (Ruconest) was produced from the milk of transgenic rabbits and two plasma-derived C1-INHs (Berinert, Cinryze) underwent controlled trials to obtain marketing authorization. In 2012, an Italian network for C1-INH-HAE (ITACA) was established by physicians of 17 HAE reference centres to collect data from Italian patients and to homogenize and improve the diagnostic and therapeutic approach to the disease. Summary Although there is a widespread agreement on therapeutic goals and treatment of C1-INH-HAE acute attacks, different approaches to prophylaxis are still present among HAE experts. The clinical experience of ITACA on a large population of C1-INH-HAE patients followed for several years may help in identifying the most effective strategies for the management of the disease. Copyright © 2015 Wolters Kluwer Health, Inc. All rights reserved.