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Long-Term Efficacy of Subcutaneous C1 Inhibitor in Pediatric Patients with Hereditary Angioedema

Authors :
Avner Reshef
Teresa Caballero
John Anderson
Ingo Pragst
Subhransu Prusty
Lawrence B. Schwartz
Henrike Feuersenger
Marco Cicardi
James R. Baker
Michael E. Manning
Donald Levy
I. Hussain
Source :
Pediatric Allergy, Immunology, and Pulmonology
Publication Year :
2020
Publisher :
Mary Ann Liebert, Inc., publishers, 2020.

Abstract

Background: Hereditary angioedema (HAE) due to C1 inhibitor (C1INH) deficiency is characterized by recurrent attacks of edema of the skin and mucosal tissues. Symptoms usually present during childhood (mean age at first attack, 10 years). Earlier symptom onset may predict a more severe disease course. Subcutaneous (SC) C1INH is indicated for routine prophylaxis to prevent HAE attacks in adolescents and adults. We analyzed the long-term efficacy of C1INH (SC) in subjects ≤17 years old treated in an open-label extension (OLE) of the pivotal phase III Clinical Study for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1 Inhibitor Replacement Therapy (COMPACT) trial. Methods: Eligible subjects (age ≥6 years, with ≥4 attacks over 2 consecutive months before entry into the OLE or placebo-controlled COMPACT trial) were treated with C1INH (SC) 40 or 60 IU/kg twice weekly for 52-140 weeks. Subgroup analyses by age (≤17 vs. >17 years) were performed for key efficacy endpoints. Results: Ten subjects were ≤17 years old [mean (range) age, 13.3 (8-16) years, 3 subjects

Details

Language :
English
ISSN :
21513228 and 2151321X
Volume :
33
Issue :
3
Database :
OpenAIRE
Journal :
Pediatric Allergy, Immunology, and Pulmonology
Accession number :
edsair.doi.dedup.....773fb4e4b19e8001674b1cc412cfbe6f