Real-world Effectiveness of Lanadelumab in Hereditary Angioedema: Multi-country 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 on every two weeks (Q2W) and every four weeks (Q4W) adjustments on AFR.

Methods: A retrospective medical chart review study was conducted in 19 HAE centers and included data from HAE type I or II patients treated with lanadelumab (index treatment) in Germany, France, Greece and Austria who were ≥ 12 years of age. Data abstraction occurred 15 September 2021 to 29 June 2022. Analyses were primarily descriptive.

Results: Data from 198 patients were collected (61.6% female, 91.9% type I HAE). Lanadelumab treatment patterns varied between countries. Cumulative AFR improved from 0% (pre-index) to 54.4% (12 months post-index) and 39.4% (post-index; median 28.8 months duration). Monthly AFRs varied from 16.2% to 28.3% pre-index (17.7% AFR in the month before index date), and from 82.7% (month 1) to >95% at multiple timepoints between 26- and 43-months post-index. Patients with interval increases (n=144, 72.7%) showed improved cumulative AFR (0% pre-index to 50.0% post-index).

Conclusions: This real-world study demonstrates that lanadelumab LTP is effective in improving AFR in HAE type I/II patients on Q2W and dose interval increases. Effectiveness with lanadelumab is rapid and was observed starting from the first month of starting therapy.