Lanadelumab demonstrates high efficacy in reducing the frequency of angioedema attacks in patients with severe HAE in real-life settings-Reference-Cited by-同舟云学术

Lanadelumab demonstrates high efficacy in reducing the frequency of angioedema attacks in patients with severe HAE in real-life settings

Published:2023-12-29 Issue:4 Volume:19 Page:334-342
ISSN:1734-1531
Container-title:Pediatria i Medycyna Rodzinna
language:
Short-container-title:Pediatr Med Rodz

Author:

Kucharczyk Aleksandra¹^ORCID,Porębski Grzegorz²^ORCID,Rząd Michał¹^ORCID,Grzela Katarzyna³^ORCID,Juchacz Aldona⁴^ORCID,Kurowski Marcin⁵^ORCID,Kuziemski Krzysztof⁶^ORCID,Łukaszyk Mateusz⁷^ORCID,Matuszewski Tomasz¹,Pawlukiewicz Małgorzata⁸^ORCID,Pawłowicz Robert⁹^ORCID,Sokołowska Małgorzata¹⁰^ORCID,Stobiecki Marcin²^ORCID,Tomasiak-Łozowska Maria¹¹^ORCID,Trębas-Pietraś Ewa¹²,Tykwińska Marta¹³^ORCID,Zakrzewska Magdalena¹⁴^ORCID,Zelent Anna¹⁵,Walczak Mieczysław¹⁶^ORCID,Jahnz-Różyk Karina¹^ORCID

Affiliation:

1. Department of Internal Diseases, Pneumonology, Allergology and Clinical Immunology, Military Institute of Medicine – National Research Institute, Warsaw, Poland

2. Department of Clinical and Environmental Allergology, Jagiellonian University Medical College, Krakow, Poland

3. Department of Paediatric Pneumonology and Allergy, Medical University of Warsaw, Warsaw, Poland

4. Wielkopolska Centre of Pulmonology and Thoracic Surgery, Poznań, Poland

5. Department of Immunology and Allergy, Chair of Pulmonology, Rheumatology and Clinical Immunology, Central Teaching Hospital, Medical University of Lodz, Łódź, Poland

6. Division of Pulmonology, Faculty of Medicine, Medical University of Gdańsk, Gdańsk, Poland

7. 1st Department of Lung Diseases and Tuberculosis, Medical University of Bialystok, Białystok, Poland

8. Provincial Clinical Hospital No. 1, Rzeszów, Poland

9. Clinical Department of Internal Medicine, Pneumonology and Allergology, Wroclaw Medical University, Wrocław, Poland

10. Department of Clinical Allergology, Pomeranian Medical University, Szczecin, Poland

11. Clinical Department of Allergy and Internal Diseases, Medical University of Bialystok, Białystok, Poland

12. Department of Allergology and Lung Diseases, Cardinal Wyszyński Provincial Specialist Hospital SPZOZ, Lublin, Poland

13. Department of Allergology, Clinical Immunology and Internal Medicine, University Hospital No. 2, Bydgoszcz, Poland

14. Department of Allergology and Pulmonology, The Warmia and Mazury Center for Lung Diseases, Olsztyn, Poland

15. Department of Pulmonology, Paediatric Allergy and Clinical Immunology, University of Medical Sciences, Poznań, Poland

16. Department of Paediatrics, Endocrinology, Diabetology, Metabolic Disease and Cardiology of the Development Age, Faculty of Medicine and Dentistry, Pomeranian Medical University, Szczecin, Poland

Abstract

Introduction and objective: Evaluation of lanadelumab efficacy in preventing angioedema attacks in patients with severe hereditary angioedema due to C1-inhibitor deficiency in Poland and descriptive analysis of this group of patients. Materials and methods: Retrospective analysis of patients treated with lanadelumab in Poland. Data were acquired from the electronic database of the National Health Fund, compiled from 15 hereditary angioedema centres. Only patients with severe hereditary angioedema course (at least 12 severe – abdominal, pharyngeal or laryngeal – hereditary angioedema attacks per six months, requiring on-demand medications) initiated treatment. The patients received lanadelumab 300 mg every two weeks. The efficacy of the therapy was assessed after six months. Results: Lanadelumab was initiated in a total of 43 patients (group B). Twenty of them achieved the follow-up point after six months (group A). The mean age of the patients was 44 years. The majority (76.7%) were female and 79% had a family history of hereditary angioedema. Most patients (95.3%) had HAE-1 (absolute deficiency of C1-inhibitor). On average, within six months before treatment, group A patients experienced 19.7 (95% confidence interval, CI: 16.06–23.33) severe hereditary angioedema attacks. In the six months following treatment initiation, the number of attacks decreased to an average of 0.5 (95% CI: 0–1.0), with significant reductions in all types of hereditary angioedema attacks – abdominal (p < 0.0001), pharyngeal (p < 0.005), and laryngeal (p < 0.05). Utilisation of on-demand medications dropped from an average of 23.5 (95% CI: 16.7–30.3) to 0.5 standard therapeutic dose (95% CI: 0–1.1). Conclusions: The study highlights the therapeutic potential of lanadelumab in managing hereditary angioedema, usually offering patients a complete resolution of severe hereditary angioedema attacks and release from dependence on rescue medication. Our results support the current paradigm shift in hereditary angioedema treatment.

Publisher

Medical Communications Sp. z.o.o.

Reference23 articles.

1. Lumry WR, Settipane RA: Hereditary angioedema: epidemiology and burden of disease. Allergy Asthma Proc 2020; 41 (Suppl 1): S8–S13.

2. Zuraw BL, Christiansen SC: HAE pathophysiology and underlying mechanisms. Clin Rev Allergy Immunol 2016; 51: 216–229.

3. Bork K, Meng G, Staubach P et al.: Hereditary angioedema: new findings concerning symptoms, affected organs, and course. Am J Med 2006; 119: 267–274.

4. Minafra FG, Gonçalves TR, Alves TM et al.: The mortality from hereditary angioedema worldwide: a review of the real-world data literature. Clin Rev Allergy Immunol 2022; 62: 232–239.

5. Longhurst H, Bygum A: The humanistic, societal, and pharmaco-economic burden of angioedema. Clin Rev Allergy Immunol 2016; 51: 230–239.

Cited by 1 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Hereditary C1-inhibitor deficiency angioedema (C1-INH-HAE) in children – practical considerations;Pediatria i Medycyna Rodzinna;2023-12-29