Preventing hereditary angioedema attacks in children using Cinryze®: interim efficacy and safety phase 3 findings

  • Background: Hereditary angioedema (HAE) is a rare genetic disease causing unpredictable and potentially life-threatening subcutaneous and submucosal edematous attacks. Cinryze® (Shire ViroPharma Inc., Lexington, MA, USA), a nanofiltered C1 inhibitor (C1-INH), is approved in Europe for the treatment, preprocedure prevention, and routine prophylaxis of HAE attacks, and for the routine prophylaxis of attacks in the USA. This phase 3 study assessed the safety and efficacy of 2 C1-INH doses in preventing attacks in children aged 6-11 years. Methods: A randomized single-blind crossover study was initiated in March 2014. Results for the first 6 patients completing the study are reported here. After a 12-week qualifying observation period, patients were randomly assigned to 1 of 2 C1-INH doses, 500 or 1,000 U, every 3-4 days for 12 weeks and crossed over to the alternative dose for a second 12-week period. The primary efficacy endpoint was the number of angioedema attacks per month. Results: Six females with HAE type I and a median age of 10.5 years received 2 doses of C1-INH (500 and 1,000 U). The mean (SD) difference in the number of monthly angioedema attacks between the baseline observation period and the treatment period was -1.89 (1.31) with 500 U and -1.89 (1.11) with 1,000 U. During the treatment periods, cumulative attack severity, cumulative daily severity, and the number of attacks needing acute treatment were lower. No serious adverse events or study drug discontinuations occurred. Conclusions: Interim findings from this study indicate that routine prevention with intravenous administration of C1-INH is efficacious, safe, and well tolerated in children ≥6 years of age.

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Author:Emel Aygören-PürsünORCiDGND, Daniel Soteres, Dumitru Moldovan, Jim Christensen, Arthur Van Leerberghe, James Hao, Jennifer Schranz, Kraig W. Jacobson, Inmaculada Martinez SaguerGND
URN:urn:nbn:de:hebis:30:3-438569
DOI:https://doi.org/10.1159/000477541
Pubmed Id:https://pubmed.ncbi.nlm.nih.gov/28662509
Parent Title (German):International archives of allergy and immunology
Publisher:Karger
Place of publication:Basel
Document Type:Article
Language:English
Year of Completion:2017
Date of first Publication:2017/06/30
Publishing Institution:Universitätsbibliothek Johann Christian Senckenberg
Release Date:2017/09/14
Tag:Attack; Cinryze®; Efficacy; Hereditary angioedema; Pediatric patients; Prevention; Safety
Volume:173
Page Number:6
First Page:114
Last Page:119
Note:
This article is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).
HeBIS-PPN:427954444
Institutes:Medizin / Medizin
Dewey Decimal Classification:6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Sammlungen:Universitätspublikationen
Licence (German):License LogoCreative Commons - Namensnennung-Nicht kommerziell - Keine Bearbeitung 4.0