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Long-Term Efficacy of Subcutaneous C1 Inhibitor in Pediatric Patients with Hereditary Angioedema
- 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
- Subjects :
- Pulmonary and Respiratory Medicine
safety
Male
Pediatrics
medicine.medical_specialty
Time Factors
Adolescent
Injections, Subcutaneous
Haegarda
Severe disease
Severity of Illness Index
Drug Administration Schedule
C1-inhibitor
03 medical and health sciences
0302 clinical medicine
Quality of life
children
030225 pediatrics
Edema
Immunology and Allergy
Medicine
Humans
Adverse effect
Child
COMPACT
Original Research
C1 inhibitor
Angioedema
biology
Dose-Response Relationship, Drug
business.industry
Angioedemas, Hereditary
medicine.disease
Optimal management
hereditary angioedema
pediatric
Treatment Outcome
030228 respiratory system
quality of life
Pediatrics, Perinatology and Child Health
Hereditary angioedema
biology.protein
Disease Progression
long term
Female
prophylaxis
medicine.symptom
business
Complement C1 Inhibitor Protein
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