Real-world Effectiveness of Lanadelumab in Hereditary Angioedema: Multi-country INTEGRATED Observational Study.

Autor:	Magerl M; Institute of Allergology, Charité - Universitätsmedizin Berlin and Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Immunology and Allergology. Electronic address: markus.magerl@charite.de., Bouillet L; Department of Internal Medicine, National Reference Centre for Angioedema (CREAK), Université Grenoble Alpes, Grenoble, France., Martinez-Saguer I; Hemophilia Centre Rhine Main (HZRM), Frankfurt, Germany., Gavini F; EUCAN Medical Affairs, Takeda Pharmaceuticals International AG, Glattpark-Opfikon (Zürich), Switzerland., Bent-Ennakhil N; EUCAN Medical Affairs, Takeda Pharmaceuticals International AG, Glattpark-Opfikon (Zürich), Switzerland., Sayegh L; Real-World Evidence, PPD, part of Thermo Fisher Scientific, Montreal, Quebec, Canada., Andresen I; EUCAN Medical Affairs, Takeda Pharmaceuticals International AG, Glattpark-Opfikon (Zürich), Switzerland.
Jazyk:	angličtina
Zdroj:	The journal of allergy and clinical immunology. In practice [J Allergy Clin Immunol Pract] 2024 Dec 17. Date of Electronic Publication: 2024 Dec 17.
DOI:	10.1016/j.jaip.2024.12.008
Abstrakt:	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. (Copyright © 2024. Published by Elsevier Inc.)
Databáze:	MEDLINE
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