Real-World Effectiveness of Lanadelumab in Hereditary Angioedema: Multicountry INTEGRATED Observational Study

BACKGROUND: Hereditary angioedema (HAE) is a rare genetic disease characterized by recurrent episodes of cutaneous or subcutaneous edema. There is clinical need for treatments that reduce the rate of HAE attacks in patients. OBJECTIVES: Primary objectives were to evaluate the effectiveness of lanadelumab on attack-free rate (AFR; proportion of patients who had zero HAE attacks), and of every 2-week and every 4-week adjustments on AFR. METHODS: A retrospective medical chart review study was conducted in 19 HAE centers and included data from patients with type I or II HAE treated with lanadelumab (index treatment) in Germany, France, Greece, and Austria who were aged 12 years or older (ClinicalTrials.gov identifier: NCT04861090). Data abstraction occurred September 15, 2021, to June 29, 2022. Analyses were primarily descriptive. RESULTS: Data from 198 patients were collected (61.6% female, 91.9% with type I HAE). Lanadelumab treatment patterns varied between countries. Cumulative AFR improved from 0% (preindex) to 54.4% (12 months postindex) and 39.4% (postindex; median duration, 28.8 months). Monthly AFRs varied from 16.2% to 28.3% preindex (17.7% AFR in the month before index date), and from 82.7% (month 1) to more than 95% at multiple time points between 26 and 43 months postindex. Patients with interval increases (n [ 144 [72.7%]) showed improved cumulative AFR (0% preindex to 50.0% postindex). CONCLUSIONS: This real-world study demonstrates that lanadelumab long-term prophylaxis is effective in improving AFR in patients with type I/II HAE on every 2-week dosing and dose interval increases. Effectiveness with lanadelumab is rapid and was observed starting from the first month of starting therapy. (c) 2024 The Authors. 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/). (J Allergy Clin Immunol Pract 2025;13:378-87)