TY  - JOUR
A1  - Aygören-Pürsün, Emel
A1  - Soteres, Daniel
A1  - Moldovan, Dumitru
A1  - Christensen, Jim
A1  - Van Leerberghe, Arthur
A1  - Hao, James
A1  - Schranz, Jennifer
A1  - Jacobson, Kraig W.
A1  - Martinez Saguer, Inmaculada
T1  - Preventing hereditary angioedema attacks in children using Cinryze®: interim efficacy and safety phase 3 findings
T2  - International archives of allergy and immunology
N2  - 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.
KW  - Cinryze®
KW  - Hereditary angioedema
KW  - Attack
KW  - Prevention
KW  - Pediatric patients
KW  - Efficacy
KW  - Safety
Y1  - 2017
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/43856
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-438569
N1  - This article is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).
VL  - 173
SP  - 114
EP  - 119
PB  - Karger
CY  - Basel
ER  -