Markus Magerl, Laurence Bouillet, Inmaculada Martinez-Saguer, Francois Gavini, Nawal Bent-Ennakhil, Laura Sayegh, Irmgard Andresen
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引用次数: 0
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 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.
期刊介绍：
JACI: In Practice is an official publication of the American Academy of Allergy, Asthma & Immunology (AAAAI). It is a companion title to The Journal of Allergy and Clinical Immunology, and it aims to provide timely clinical papers, case reports, and management recommendations to clinical allergists and other physicians dealing with allergic and immunologic diseases in their practice. The mission of JACI: In Practice is to offer valid and impactful information that supports evidence-based clinical decisions in the diagnosis and management of asthma, allergies, immunologic conditions, and related diseases.
This journal publishes articles on various conditions treated by allergist-immunologists, including food allergy, respiratory disorders (such as asthma, rhinitis, nasal polyps, sinusitis, cough, ABPA, and hypersensitivity pneumonitis), drug allergy, insect sting allergy, anaphylaxis, dermatologic disorders (such as atopic dermatitis, contact dermatitis, urticaria, angioedema, and HAE), immunodeficiency, autoinflammatory syndromes, eosinophilic disorders, and mast cell disorders.
The focus of the journal is on providing cutting-edge clinical information that practitioners can use in their everyday practice or to acquire new knowledge and skills for the benefit of their patients. However, mechanistic or translational studies without immediate or near future clinical relevance, as well as animal studies, are not within the scope of the journal.
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