title: Replacement therapy with C1 esterase inhibitors for hereditary angioedema.
abstract: Angioedema due to hereditary C1 inhibitor deficiency (HAE) is a highly disabling and potentially lethal disease with an estimated prevalence of 1:50,000 in the general population worldwide. Treatment of this condition by replacing the deficient protein started shortly after discovery of the underlying genetic defect. Along with other therapeutic approaches developed over the years, C1 inhibitor replacement therapy maintains a central role for the treatment of angioedema attacks in patients with HAE. Two plasma-derived C1 inhibitors and a recombinant form, produced in transgenic rabbits, have successfully completed controlled trials that reinforced the evidence of the safety and efficacy of this treatment.
authors.affiliation: Dipartimento di Scienze Cliniche "Luigi Sacco" Università di Milano, Ospedale L. Sacco, Milano, Italy. marco.cicardi@unimi.it
authors.forename: M
authors.initials: M
authors.lastname: Cicardi
authors.forename: A
authors.initials: A
authors.lastname: Zanichelli
mesh_terms: D054179:Angioedemas, Hereditary / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology; D000818:Animals; D030801:Animals, Genetically Modified; D003174:Complement C1 Inactivator Proteins / Q000009:adverse effects / Q000627:therapeutic use*; D018849:Controlled Clinical Trials as Topic; D015195:Drug Design; D006801:Humans; D011817:Rabbits
chemical_list: D003174:Complement C1 Inactivator Proteins