• Marc A Riedl, David S Hurewitz, Robyn Levy, Paula J Busse, David Fitts, and Ira Kalfus.
• Department of Clinical Immunology and Allergy, David Geffen School of Medicine, University of California, Los Angeles, California. Electronic address: mriedl@mednet.ucla.edu.
• Ann. Allergy Asthma Immunol. 2012 Jan 1; 108 (1): 49-53.

BackgroundHereditary angioedema (HAE) is a rare disease caused by C1INH gene mutations, which leads to a deficiency or dysfunction of C1 inhibitor (C1 INH), resulting in recurrent episodes of severe and potentially life-threatening edema.ObjectiveTo evaluate the efficacy and safety of repeat use of nanofiltered C1 esterase inhibitor (human) (C1 INH-nf) for the short-term treatment of HAE attacks.MethodsIn this open-label study, patients received C1 INH-nf, 1,000 U intravenously, for the treatment of HAE attacks. Efficacy end points included the proportion of attacks with unequivocal relief of the defining symptom within 1 and 4 hours after receiving study drug and time to beginning of relief of the defining symptom. Safety was monitored through adverse event reporting, vital signs measurements, and laboratory testing.ResultsA total of 113 patients were enrolled in the study from September 21, 2006, through March 31, 2009, and received 885 doses of C1 INH-nf. A total of 609 HAE attacks were treated with C1 INH-nf, and the numbers of attacks achieving unequivocal relief of the defining symptom within 1 and 4 hours after the start of the first dose were 412 (68%) and 529 (87%), respectively. Of 101 patients treated for an attack during the study period, 80 achieved unequivocal relief of their first attack within 4 hours after study medication (response rate, 79%); median time to the beginning of unequivocal relief was 0.75 hour. C1 INH-nf was safe and well tolerated.ConclusionsThis open-label study demonstrates the efficacy and safety of C1 INH-nf for short-term treatment of HAE attacks.Trial Registrationclinicaltrials.gov Identifier: NCT00438815.Copyright © 2012 American College of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved.

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