Buckland, Matthew S;
Boccon-Gibod, Isabelle;
De Moreuil, Claire;
Sanges, Sébastien;
(2025)
Berotralstat effectiveness and safety in patients with hereditary angioedema with normal C1-inhibitor.
Journal of Allergy and Clinical Immunology: Global
, 5
(1)
, Article 100575. 10.1016/j.jacig.2025.100575.
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Abstract
Background: Hereditary angioedema (HAE) with normal C1 inhibitor (nC1-INH) is the least common endotype of HAE, a rare disorder with localized, intermittent attacks of soft tissue swelling. Prophylactic treatments are available across HAE endotypes, including berotralstat, a once-daily oral inhibitor of plasma kallikrein. Objective: This European multicenter case series aimed to report treatment-related outcomes with berotralstat in patients with HAE-nC1-INH. Methods: A retrospective case series analysis including observational data from patients in United Kingdom and French centers was performed. Patients were included if they had a genetic assay demonstrating a known HAE-nC1-INH–associated variant; or had a family history of angioedema and normal C1-INH level and function, no response to antihistamine treatment, met predefined diagnostic criteria for HAE-nC1-INH, and had been prescribed berotralstat 150 mg daily for at least 6 months. Data were collected from the patients’ records using a standardized form. Results: Four female and 2 male patients with HAE-nC1-INH were included from 4 centers in France and the United Kingdom. Duration of berotralstat treatment at analysis ranged from 6 to 23 months. Five patients showed a response to berotralstat, observed as a 29-100% reduction in attack rates. Three patients experienced a reduction in attack severity from moderate to minor or mild. One patient reported adverse events during berotralstat initiation: nausea and diarrhea. Conclusion: Long-term prophylactic berotralstat was effective in reducing HAE attacks for 5 of 6 patients with HAE-nC1-INH. No significant safety signals were noted. (J Allergy Clin Immunol Global 2026;5:100575.
| Type: | Article |
|---|---|
| Title: | Berotralstat effectiveness and safety in patients with hereditary angioedema with normal C1-inhibitor |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.jacig.2025.100575 |
| Publisher version: | https://doi.org/10.1016/j.jacig.2025.100575 |
| Language: | English |
| Additional information: | © 2025 The Author(s). Published by Elsevier Inc. on behalf of the American Academy of Allergy, Asthma & Immunology. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/) |
| Keywords: | Angioedema, berotralstat, bradykinin, C1 inhibitor, hereditary, kallikrein |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10214946 |
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