Trial reportPediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology2019
A randomized trial of human C1 inhibitor prophylaxis in children with hereditary angioedema.
Trial report in Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02052141 (A Phase 3, Multicenter, Randomized, Single-Blind, Dose-Ranging, Crossover Study to Evaluate the Safety and Efficacy of Intravenous Administration of CINRYZE®), which is not on this map. Cited by 16 papers, 4 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 3, Multicenter, Randomized, Single-Blind, Dose-Ranging, Crossover Study to Evaluate the Safety and Efficacy of Intravenous Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) for the Prevention of Angioedema Attacks in Children 6 to 11 Years of Age With Hereditary Angioedema
Who cites it
16 citing papers in PubMed, 4 syntheses or guidelines pooled it, 29 citations in OpenAlex.
- International Guideline on the Diagnosis and Management of Pediatric Patients With Hereditary Angioedema.Allergy · 2026Guideline
- Indirect treatment comparison of lanadelumab and a C1-esterase inhibitor in pediatric patients with hereditary angioedema.Journal of comparative effectiveness research · 2025Pooled it
- Hereditary Angioedema Attacks in Patients Receiving Long-Term Prophylaxis: A Systematic Review.Clinical reviews in allergy & immunology · 2024Pooled it
- Interventions for the long-term prevention of hereditary angioedema attacks.The Cochrane database of systematic reviews · 2022Pooled it
- A randomized trial of human C1 inhibitor prophylaxis in children with hereditary angioedema.Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology · 2019Trial
- Sebetralstat for breakthrough attacks in patients with hereditary angioedema receiving long-term prophylaxis in KONFIDENT-S.The journal of allergy and clinical immunology. Global · 2026Article
- The 2025 WAO Guidelines for the classification, diagnosis, and treatment of hereditary angioedema, with consideration of worldwide disparities.The World Allergy Organization journal · 2026Article
- The International/Canadian hereditary angioedema guideline.Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology · 2026Review
- [Hereditary angioedema due to C1-inhibitor deficiency, a national disease management programme].Wiener klinische Wochenschrift · 2023Article
- The estimation of health state utility values in rare diseases: do the approaches in submissions for NICE technology appraisals reflect the existing literature? A scoping review.The European journal of health economics : HEPAC : health economics in prevention and care · 2023Article
- Current challenges and future opportunities in patient-focused management of hereditary angioedema: A narrative review.Clinical and translational allergy · 2023Review
- A Review of Randomized Controlled Trials of Hereditary Angioedema Long-Term Prophylaxis with C1 Inhibitor Replacement Therapy: Alleviation of Disease Symptoms Is Achievable.Journal of asthma and allergy · 2023Review
- The remarkably frequent use of EQ-5D in non-economic research.The European journal of health economics : HEPAC : health economics in prevention and care · 2022Article
- The international WAO/EAACI guideline for the management of hereditary angioedema - The 2021 revision and update.The World Allergy Organization journal · 2022Article
- Racial and Ethnic Disparities in the Research and Care of Hereditary Angioedema Patients in the United States.The journal of allergy and clinical immunology. In practice · 2021Article
- Assessment and management of disease burden and quality of life in patients with hereditary angioedema: a consensus report.Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 4 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPatients 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.
methodsEligible 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).
resultsTwelve 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.
conclusionsC1-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 registrationClinicalTrials.gov identifier NCT02052141.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.