Trial reportPediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology2019
Recombinant human C1 esterase inhibitor treatment for hereditary angioedema attacks in children.
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 NCT01359969 (Open-label, Phase II, Single Arm Study to Evaluate the Safety, Immunogenicity, Pharmacokinetics and Efficacy of rhC1INH for the Treatment of Acute Attacks in Pediatric Patients With Hereditary Angioedema, From 2-13 Years of Age), which is not on this map. Cited by 16 papers, 3 of them syntheses that pooled it.
What it found
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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.
Open-label, Phase II, Single Arm Study to Evaluate the Safety, Immunogenicity, Pharmacokinetics and Efficacy of rhC1INH for the Treatment of Acute Attacks in Pediatric Patients With Hereditary Angioedema, From 2-13 Years of Age
Who cites it
16 citing papers in PubMed, 3 syntheses or guidelines pooled it, 24 citations in OpenAlex.
- Diverse Trial Designs, Populations, and Outcomes: A Systematic Literature Review of Trials for the Treatment of Hereditary Angioedema Attacks.Advances in therapy · 2026Pooled it
- 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
- Recombinant Human C1 Esterase Inhibitor for the Management of Adverse Events Related to Intravenous Immunoglobulin Infusion in Patients With Common Variable Immunodeficiency or Polyneuropathy: A Pilot Open-Label Study.Frontiers in immunology · 2021Trial
- Recombinant human C1 esterase inhibitor treatment for hereditary angioedema attacks in children.Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology · 2019Trial
- 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 (HAE) in children and adolescents: New treatment options.Allergologie select · 2024Review
- Epidemiology and treatment of children with hereditary angioedema in Germany: A retrospective database study.Clinical and translational allergy · 2023Article
- The international WAO/EAACI guideline for the management of hereditary angioedema - The 2021 revision and update.The World Allergy Organization journal · 2022Article
- Clinical profile and treatment outcomes in patients with hereditary angioedema with normal C1 esterase inhibitor.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
- Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry.The World Allergy Organization journal · 2021Article
- Therapeutic management of hereditary angioedema: past, present, and future.Balkan medical journal · 2021Article
- Hereditary Angioedema Attack in Utero and Treatment of the Mother and Fetus.Mayo Clinic proceedings. Innovations, quality & outcomes · 2020Article
- Modeling Cost-Effectiveness of On-Demand Treatment for Hereditary Angioedema Attacks.Journal of managed care & specialty pharmacy · 2020Article
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 6 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundAttacks of hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency (C1-INH-HAE) usually begin during childhood or adolescence. However, limited data are available regarding indications and modalities of treatment of children. This study evaluated recombinant human C1-INH (rhC1-INH) for HAE attacks in children.
methodsThis open-label, phase 2 study included children aged 2-13 years with C1-INH-HAE. Eligible HAE attacks were treated intravenously with rhC1-INH 50 IU/kg body weight (maximum, 4200 IU). The primary end-point was time to beginning of symptom relief (TOSR; ≥20 mm decrease from baseline in visual analog scale [VAS] score, persisting for two consecutive assessments); secondary end-point was time to minimal symptoms (TTMS; <20 mm VAS score for all anatomic locations).
resultsTwenty children (aged 5-14 years; 73 HAE attacks) were treated with rhC1-INH. Seventy (95.9%) of the attacks were treated with a single dose of rhC1-INH. Seven (35.0%) children were treated for four or more attacks. Overall, median TOSR was 60.0 minutes (95% confidence interval [CI], 60.0-65.0); data were consistent across attacks. Median TTMS was 122.5 minutes (95% CI, 120.0-126.0); data were consistent across attacks. No children withdrew from the study due to adverse events. No treatment-related serious adverse events or hypersensitivity reactions were reported; no neutralizing antibodies were detected.
conclusionsRecombinant human C1-INH was efficacious, safe, and well tolerated in children. Data support use of the same dosing regimen for HAE attacks in children (50 IU/kg; up to 4200 IU, followed by an additional dose, if needed) as is currently recommended for adolescents and adults.
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Registered trials
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.