ArticleFrontiers in immunology2024
Berotralstat in hereditary angioedema due to C1 inhibitor deficiency: first real-world evidence from a Canadian center.
Article in Frontiers in immunology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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Who cites it
6 citing papers in PubMed.
- Attack rate reductions following berotralstat initiation among US patients with hereditary angioedema in the real-world.Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology · 2026Article
- Hereditary Angioedema Prophylaxis Therapy: Berotralstat and Lanadelumab Safety Profile.Medicina (Kaunas, Lithuania) · 2025Article
- Reductions in medical visits and hospitalizations following berotralstat initiation in patients with hereditary angioedema in the United States.Journal of managed care & specialty pharmacy · 2025Article
- Disease control and disease activity in hereditary angioedema: two sides of the same coin?Frontiers in immunology · 2025Article
- Berotralstat for hereditary angioedema with C1 inhibitor deficiency: a practical guide for clinicians.Frontiers in immunology · 2024Article
- Centralized care model for hereditary angioedema overcomes geographical barriers.Frontiers in immunology · 2024Article
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Authors and funding
3 authors.
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Abstract
Background: Hereditary angioedema due to C1 inhibitor deficiency is a rare genetic condition that causes recurrent swelling with consequent functional impairment and decreased quality of life. Long-term prophylaxis (LTP) to prevent angioedema episodes is a key component of disease management. Berotralstat, an oral, once-daily plasma kallikrein inhibitor, was approved for LTP by Health Canada in 2022. Methods: We conducted a retrospective, real-world study investigating the effectiveness and adverse effects of berotralstat. Data on angioedema frequency, disease control, and adverse events were tabulated. Patient satisfaction with treatment was scored on a 5-point Likert scale, with 1 representing very unsatisfied and 5 representing very satisfied with therapy. Results: From June, 2022 and May, 2023, 8 patients with HAE type 1 or type 2 received berotralstat. Effectiveness data were available for 7 patients who continued the drug for at least 3 months, 4 of whom switched to berotralstat from plasma-derived C1 inhibitor LTP. In these 7 patients, the average number of attacks per month decreased from 3.3 to 1.6 (p<0.05), representing a ~52% reduction in attack frequency. Median angioedema control test score numerically improved from 8 to 13 (p=0.0781). Of the 8 patients who received berotralstat, 3 reported no adverse effects and 5 experienced gastrointestinal side effects, which were mild and transient in 3 and led to discontinuation in 1. Average treatment satisfaction was between satisfied and very satisfied at 4.3. Conclusion: Berotralstat is an effective agent for long-term prophylaxis in HAE. Most patients experienced no adverse effects or mild, transient gastrointestinal symptoms.
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