Related Experiment Video
Updated: Jan 26, 2026
Author Spotlight: Advancements and Challenges in Surgical Treatments for Postamputation Pain
Published on: March 8, 2024
A randomized trial of human C1 inhibitor prophylaxis in children with hereditary angioedema
Emel Aygören-Pürsün1, Daniel F Soteres2, Sandra A Nieto-Martinez3
1Department for Children and Adolescents, Angioedema Centre, University Hospital Frankfurt, Goethe University, Frankfurt, Germany.
Insights
C1 inhibitor prophylaxis significantly reduced angioedema attacks in children aged 6-11 years. Treatment was safe, well-tolerated, and improved health-related quality of life (HRQoL).
Area of Science:
- Immunology
- Genetics
- Pediatrics
Background:
- Hereditary angioedema (HAE) with C1 inhibitor deficiency causes recurrent attacks.
- Current treatments for HAE in children aged 6-11 years require investigation for safety and efficacy.
Purpose of the Study:
- To investigate the safety, efficacy, and health-related quality of life (HRQoL) of C1 inhibitor prophylaxis in children with HAE.
- To compare two doses of intravenously administered C1 inhibitor (C1-INH) in pediatric patients.
Main Methods:
- A randomized, single-blind, crossover, phase 3 trial.
- Patients received 500 U or 1000 U C1-INH twice weekly for 12 weeks, followed by crossover to the alternate dose.
- Primary endpoint was the monthly normalized number of angioedema attacks (NNA); HRQoL assessed via EQ-5D-Y and EQ-VAS.
Main Results:
- Both 500 U and 1000 U C1-INH significantly reduced monthly NNA compared to baseline (71.1% and 84.5% reduction, respectively).
- Within-patient analysis showed a significant reduction in NNA with both doses (P=0.035).
- Improvements in HRQoL, measured by EQ-VAS, exceeded the minimal important difference, indicating meaningful patient benefit. No serious adverse events were reported.
Conclusions:
- C1-INH prophylaxis is effective and safe for managing recurrent angioedema attacks in children aged 6-11 years.
- Intravenous C1-INH treatment leads to significant reductions in attack frequency and severity.
- The treatment demonstrates a positive impact on the health-related quality of life for pediatric patients with HAE.
Background:
Patients with hereditary angioedema with C1 inhibitor deficiency or dysfunction have burdensome recurrent angioedema attacks. The safety, efficacy, and health-related quality of life (HRQoL) outcomes of C1 inhibitor (C1-INH) prophylaxis (intravenously administered) in patients aged 6-11 years were investigated.
Methods:
Eligible patients were enrolled in a randomized, single-blind, crossover, phase 3 trial. After a 12-week baseline observation period (BOP), patients received 500 or 1000 U C1-INH, twice weekly, for 12 weeks before crossing over to the alternate dose for 12 weeks. The primary efficacy end-point was the monthly normalized number of angioedema attacks (NNA). HRQoL was assessed using the EuroQoL 5-dimensional descriptive system youth version and visual analog scale (EQ-VAS).
Results:
Twelve randomized patients had a median (range) age of 10.0 (7-11) years. Mean (SD) percentage reduction in monthly NNA from BOP was 71.1% (27.1%) with 500 U and 84.5% (20.0%) with 1000 U C1-INH. Mean (SD) within-patient difference (-0.4 [0.58]) for monthly NNA with both doses was significant (P = 0.035 [90% CI, -0.706 to -0.102]). Cumulative attack severity, cumulative daily severity, and number of acute attacks treated were reduced. No serious adverse events or discontinuations occurred. Mean EQ-VAS change from BOP to week 9 of treatment (500 U C1-INH, 10.4; 1000 U C1-INH, 21.6) was greater than the minimal important difference, indicating a meaningful HRQoL change.
Conclusions:
C1-INH prophylaxis was effective, safe, and well tolerated in children aged 6-11 years experiencing recurrent angioedema attacks. A post hoc analysis indicated a meaningful improvement in HRQoL with C1-INH.
Trial Registration:
ClinicalTrials.gov identifier NCT02052141.
More Related Videos
Related Concept Videos
01:52Eukaryotic Transcription Inhibitors
Eukaryotic transcription inhibitors usually contain two distinct domains, a...
01:16Clinical Trials
There are four phases in a clinical trial. A phase one...
01:11Clinical Trials: Overview
01:12Trial and Error and Algorithm
01:04Random Error
01:09Random Variables
Uppercase letters such as X or Y denote a random variable. Lowercase letters like x or y denote the value of a random variable. If X is a random variable, then X is written in words, and x is given as a number.
For example, let X = the...