Consensus on treatment goals in hereditary angioedema: A global Delphi initiative.

Autor: Maurer M; Angioedema Center of Reference and Excellence, Dermatological Allergology, Allergie-Centrum-Charité, Department of Dermatology and Allergy, Charité-Universitätsmedizin Berlin, Berlin, Germany. Electronic address: marcus.maurer@charite.de., Aygören-Pürsün E; Department of Children and Adolescents, University Hospital Frankfurt, Frankfurt, Germany., Banerji A; Division of Rheumatology, Allergy and Immunology, Massachusetts General Hospital, Department of Medicine, Harvard Medical School, Boston, Mass., Bernstein JA; Department of Medicine, Division of Immunology/Allergy Section, University of Cincinnati, Cincinnati, Ohio., Balle Boysen H; HAE International (HAE = Hereditary Angioedema), Horsens, Denmark., Busse PJ; Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY., Bygum A; Department of Clinical Genetics, Odense University Hospital, Odense, Denmark; Clinical Institute, University of Southern Denmark, Odense, Denmark., Caballero T; Allergy Department, Hospital Universitario La Paz, Hospital La Paz Institute for Health Research, Centre for Biomedical Network Research on Rare Diseases, Madrid, Spain., Castaldo AJ; US HAE Association, Fairfax City, Va., Christiansen SC; US HAE Association Angioedema Center, Department of Medicine, University of California, San Diego, La Jolla, Calif., Craig T; Department of Medicine, Pediatrics and Graduate Studies, Penn State University, Hershey, Pa., Farkas H; Hungarian Angioedema Center of Reference and Excellence, Department of Internal Medicine and Haematology, Semmelweis University, Budapest, Hungary., Grumach AS; Clinical Immunology, Faculdade de Medicina, Centro Universitário Saude ABC, São Paulo, Brazil., Hide M; Department of Dermatology, Hiroshima University, Hiroshima, Japan., Katelaris CH; Department of Medicine, Campbelltown Hospital and Western Sydney University, Campbelltown, Australia., Li HH; Institute for Asthma and Allergy, Chevy Chase, Md., Longhurst H; Auckland City Hospital, Auckland, New Zealand; University College Hospital, London, United Kingdom., Lumry WR; Department of Internal Medicine, Allergy/Immunology Division, Southwestern Medical School, University of Texas, Dallas, Tex; Allergy and Asthma Research Association Research Center, Dallas, Tex., Magerl M; Angioedema Center of Reference and Excellence, Dermatological Allergology, Allergie-Centrum-Charité, Department of Dermatology and Allergy, Charité-Universitätsmedizin Berlin, Berlin, Germany., Martinez-Saguer I; Hemophilia Centre Rhine Main, Mörfelden-Walldorf, Germany., Riedl MA; US HAE Association Angioedema Center, Department of Medicine, University of California, San Diego, La Jolla, Calif., Zhi Y; Department of Allergy and Clinical Immunology, Peking Union Medical College Hospital and Chinese Academy of Medical Sciences, National Clinical Research Center for Immunologic Disease, Beijing, China., Zuraw B; US HAE Association Angioedema Center, Department of Medicine, University of California, San Diego, La Jolla, Calif; San Diego Veterans Affairs Healthcare, San Diego, Calif.
Jazyk: angličtina
Zdroj: The Journal of allergy and clinical immunology [J Allergy Clin Immunol] 2021 Dec; Vol. 148 (6), pp. 1526-1532. Date of Electronic Publication: 2021 May 25.
DOI: 10.1016/j.jaci.2021.05.016
Abstrakt: Background: Hereditary angioedema (HAE) is a rare, life-threatening genetic disorder characterized by recurrent episodes of subcutaneous or submucosal angioedema. The ultimate goals of treatment for HAE remain ill-defined.
Objectives: The aim of this Delphi process was to define the goals of HAE treatment and to examine which factors should be considered when assessing disease control and normalization of the patient's life.
Methods: The Delphi panel comprised 23 participants who were selected based on involvement with scientific research on HAE or coauthorship of the most recent update and revision of the World Allergy Organization/European Academy of Allergy and Clinical Immunology guideline on HAE. The process comprised 3 rounds of voting. The final round aimed to aggregate the opinions of the expert panel and to achieve consensus.
Results: Two direct consensus questions were posed in round 2, based on the responses received in round 1, and the panel agreed that the goals of treatment are to achieve total control of the disease and to normalize the patient's life. For the third round of voting, 21 statements were considered, with the participants reaching consensus on 18. It is clear from the wide-ranging consensus statements that the burdens of disease and treatment should be considered when assessing disease control and normalization of patients' lives.
Conclusions: The ultimate goal for HAE treatment is to achieve no angioedema attacks. The availability of improved treatments and disease management over the last decade now makes complete control of HAE a realistic possibility for most patients.
(Copyright © 2021 The Authors. Published by Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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