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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.
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http://dx.doi.org/10.1016/j.jaip.2024.12.008 | DOI Listing |
Front Allergy
August 2025
Internal Medicine, George Emil Palade University of Medicine, Pharmacy, Science, and Technology of Targu Mures, Targu Mures, Romania.
Introduction: In the majority of patients with hereditary angioedema (HAE) due to C1-inhibitor deficiency (HAE-C1INH), effective long-term prophylactic (LTP) treatment can achieve complete disease control. Lanadelumab is one of the first-line option recommended for this purpose. Our study aimed to evaluate changes in disease control, quality of life, and attack frequency among Romanian HAE-C1INH patients, during the first year of treatment with lanadelumab.
View Article and Find Full Text PDFJ Allergy Clin Immunol Pract
September 2025
Department of Clinical Immunology, Centro Universitario Faculdade de Medicina do ABC, Santo André, SP, Brazil.
Background: Hereditary angioedema (HAE) is a rare autosomal dominant disorder with a prevalence of 1:50,000 individuals. Delayed diagnosis and deaths from asphyxia still occur.
Objective: To identify knowledge and management gaps regarding clinical, genetic, and therapeutic aspects of HAE in Brazil, aiming to improve patient care and outcomes.
J Clin Immunol
August 2025
Department of Allergy & Immunology, Department of Dermatology, Huashan Hospital, Fudan University, 12 Middle Urumqi Road, Shanghai, 200040, P.R. China.
Hereditary angioedema with normal C1 inhibitor (HAE-nC1-INH) is a rare and genetically heterogeneous disorder with an incomplete molecular understanding. This study aimed to identify novel genetic variants associated with HAE-nC1-INH, characterize their clinical manifestations, and evaluate real-world treatment responses. Whole-exome sequencing of 27 HAE patients, including eight with HAE-nC1-INH, identified four previously unreported MYOF variants and additional pathogenic variants in KNG1 and HS3ST6, expanding the genetic spectrum of the disease.
View Article and Find Full Text PDFOrphanet J Rare Dis
August 2025
Department of Allergy, Renji Hospital, Shanghai Jiao Tong University School of Medicine, 200001, Shanghai, China.
Background: Hereditary angioedema (HAE) is a rare monogenic disease, and there are few reports on its clinical characteristics, particularly its drug efficacy, in China. The objective of this study was to gain insight into the clinical characteristics of HAE in Chinese patients, and the efficacy and safety of prophylactic treatment with lanadelumab.
Results: The cohort included 22 patients with a median age of 35.
Ann Allergy Asthma Immunol
September 2025
Hämophilie-Zentrum Rhein Main (HZRM), Frankfurt/Main, Germany.
Background: Real-world clinical data support effectiveness and safety of lanadelumab in patients with hereditary angioedema (HAE); however, disease activity between patients can vary substantially in the absence of long-term prophylactic treatment.
Objective: To assess the effectiveness of lanadelumab in patients with HAE by baseline HAE attack frequency.
Methods: Patients with HAE from the phase 4 EMPOWER (NCT03845400) and ENABLE (NCT04130191) studies with available baseline attack rate data were included in this post hoc analysis.