Real-World Effectiveness of Lanadelumab in Hereditary Angioedema: Multicountry INTEGRATED Observational Study
Markus Magerl, Laurence Bouillet, Inmaculada Martinez‐Saguer, François Gavini, Nawal Bent‐Ennakhil, Laura Sayegh, Irmgard Andresen
Abstract
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.