ArticleAllergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology2019
Subcutaneous C1 inhibitor for prevention of attacks of hereditary angioedema: additional outcomes and subgroup analysis of a placebo-controlled randomized study.
Article in Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers, 2 of them syntheses that pooled it.
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Who cites it
12 citing papers in PubMed, 2 syntheses or guidelines pooled it, 15 citations in OpenAlex.
- 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
- Lanadelumab demonstrates rapid and sustained prevention of hereditary angioedema attacks.Allergy · 2020Trial
- Patient Preferences for Long-Term Prophylactic Treatment in Hereditary Angioedema: A Discrete-Choice Experiment.The patient · 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: on-demand treatment and long-term prophylaxis-a global reality.Clinical and experimental immunology · 2026Review
- Unraveling angioedema: diagnostic challenges and emerging therapies.Frontiers in immunology · 2025Review
- The international WAO/EAACI guideline for the management of hereditary angioedema - The 2021 revision and update.The World Allergy Organization journal · 2022Article
- A catalog of the genetic causes of hereditary angioedema in the Canary Islands (Spain).Frontiers in immunology · 2022Article
- Reviewing clinical considerations and guideline recommendations of C1 inhibitor prophylaxis for hereditary angioedema.Clinical and translational allergy · 2022Review
- First Census of Patients with Hereditary Angioedema in the Canary Islands.Journal of clinical medicine · 2021Article
Corrections and comments
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Authors and funding
17 authors at 9 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundHereditary angioedema (HAE) is a debilitating disorder resulting from C1-esterase inhibitor (C1-INH) deficiency. In the COMPACT phase 3 study the prophylactic use of a subcutaneous C1 inhibitor (C1-INH [SC], HAEGARDA
methodsThis is a post hoc exploratory analysis on data from the randomized, placebo-controlled COMPACT study. 90 patients with C1-INH-HAE were randomized to 1 of 4 treatment sequences: C1-INH (SC) 40 or 60 IU/kg of body weight twice weekly for 16 weeks, preceded or followed by a placebo period. The pre-specified primary efficacy endpoint was the time-normalized number of HAE attacks, and pre-specified secondary efficacy endpoints were the percentage of patients with a certain treatment response (≥ 50% reduction on C1-INH (SC) versus placebo in the time-normalized number of attacks) and the time-normalized number of use of rescue medication. Pre-specified exploratory endpoints included severity of attacks, alone and combined with rescue medication use. Post hoc analyses included exploration of onset of effect and clinical assessment of patients with < 50% of response.
resultsSubgroup findings by various patient characteristics showed a consistent preventive effect of C1-INH (SC). In a post hoc analysis of attacks, the onset of the preventive effect within the first 2 weeks after treatment initiation in COMPACT showed that 10/43 patients (23%) experienced attacks of any severity with 60 IU/kg versus 34/42 patients (81%) with placebo. The need for rescue medication was tenfold lower with 60 IU/kg (35 treated attacks) versus placebo (358 treated attacks). A qualitative analysis of the 4 patients treated with 60 IU/kg and with < 50% reduction of attacks demonstrated a reduction in severity of attacks, rescue medication use, and symptom days which was considered a clinically meaningful treatment effect.
conclusionsC1-INH (SC) prophylaxis demonstrated a preventive treatment effect with evidence of benefit within 2 weeks. A consistent treatment effect at recommended C1-INH (SC) dosing was evident in all subgroups of patients with type I/II HAE and by various measures of disease and treatment burden.
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