Marcus Maurer1, Emel Aygören-Pürsün2, Aleena Banerji3, Jonathan A Bernstein4, Henrik Balle Boysen5, Paula J Busse6, Anette Bygum7, Teresa Caballero8, Anthony J Castaldo9, Sandra C Christiansen10, Timothy Craig11, Henriette Farkas12, Anete S Grumach13, Michihiro Hide14, Constance H Katelaris15, H Henry Li16, Hilary Longhurst17, William R Lumry18, Markus Magerl19, Inmaculada Martinez-Saguer20, Marc A Riedl10, Yuxiang Zhi21, Bruce Zuraw22. 1. 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. 2. Department of Children and Adolescents, University Hospital Frankfurt, Frankfurt, Germany. 3. Division of Rheumatology, Allergy and Immunology, Massachusetts General Hospital, Department of Medicine, Harvard Medical School, Boston, Mass. 4. Department of Medicine, Division of Immunology/Allergy Section, University of Cincinnati, Cincinnati, Ohio. 5. HAE International (HAE = Hereditary Angioedema), Horsens, Denmark. 6. Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY. 7. Department of Clinical Genetics, Odense University Hospital, Odense, Denmark; Clinical Institute, University of Southern Denmark, Odense, Denmark. 8. Allergy Department, Hospital Universitario La Paz, Hospital La Paz Institute for Health Research, Centre for Biomedical Network Research on Rare Diseases, Madrid, Spain. 9. US HAE Association, Fairfax City, Va. 10. US HAE Association Angioedema Center, Department of Medicine, University of California, San Diego, La Jolla, Calif. 11. Department of Medicine, Pediatrics and Graduate Studies, Penn State University, Hershey, Pa. 12. Hungarian Angioedema Center of Reference and Excellence, Department of Internal Medicine and Haematology, Semmelweis University, Budapest, Hungary. 13. Clinical Immunology, Faculdade de Medicina, Centro Universitário Saude ABC, São Paulo, Brazil. 14. Department of Dermatology, Hiroshima University, Hiroshima, Japan. 15. Department of Medicine, Campbelltown Hospital and Western Sydney University, Campbelltown, Australia. 16. Institute for Asthma and Allergy, Chevy Chase, Md. 17. Auckland City Hospital, Auckland, New Zealand; University College Hospital, London, United Kingdom. 18. Department of Internal Medicine, Allergy/Immunology Division, Southwestern Medical School, University of Texas, Dallas, Tex; Allergy and Asthma Research Association Research Center, Dallas, Tex. 19. Angioedema Center of Reference and Excellence, Dermatological Allergology, Allergie-Centrum-Charité, Department of Dermatology and Allergy, Charité-Universitätsmedizin Berlin, Berlin, Germany. 20. Hemophilia Centre Rhine Main, Mörfelden-Walldorf, Germany. 21. 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. 22. US HAE Association Angioedema Center, Department of Medicine, University of California, San Diego, La Jolla, Calif; San Diego Veterans Affairs Healthcare, San Diego, Calif.
Abstract
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.
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.
Authors: Marcus Maurer; Markus Magerl; Stephen Betschel; Werner Aberer; Ignacio J Ansotegui; Emel Aygören-Pürsün; Aleena Banerji; Noémi-Anna Bara; Isabelle Boccon-Gibod; Konrad Bork; Laurence Bouillet; Henrik Balle Boysen; Nicholas Brodszki; Paula J Busse; Anette Bygum; Teresa Caballero; Mauro Cancian; Anthony J Castaldo; Danny M Cohn; Dorottya Csuka; Henriette Farkas; Mark Gompels; Richard Gower; Anete S Grumach; Guillermo Guidos-Fogelbach; Michihiro Hide; Hye-Ryun Kang; Allen P Kaplan; Constance H Katelaris; Sorena Kiani-Alikhan; Wei-Te Lei; Richard F Lockey; Hilary Longhurst; William Lumry; Andrew MacGinnitie; Alejandro Malbran; Inmaculada Martinez Saguer; Juan José Matta Campos; Alexander Nast; Dinh Nguyen; Sandra A Nieto-Martinez; Ruby Pawankar; Jonathan Peter; Grzegorz Porebski; Nieves Prior; Avner Reshef; Marc Riedl; Bruce Ritchie; Farrukh Rafique Sheikh; William B Smith; Peter J Spaeth; Marcin Stobiecki; Elias Toubi; Lilian Agnes Varga; Karsten Weller; Andrea Zanichelli; Yuxiang Zhi; Bruce Zuraw; Timothy Craig Journal: World Allergy Organ J Date: 2022-04-07 Impact factor: 5.516
Authors: Natalia Gabriel; Fernanda Marcelino; Mariana P L Ferriani; L Karla Arruda; Regis A Campos; Rozana F Gonçalves; Herberto Chong-Neto; Nelson Rosario Filho; Solange O R Valle; Joao B Pesquero; Anete S Grumach Journal: Front Allergy Date: 2022-02-17
Authors: P Triggianese; R Senter; A Petraroli; A Zoli; M Lo Pizzo; D Bignardi; E Di Agosta; S Agolini; F Arcoleo; O Rossi; S Modica; E Greco; M S Chimenti; G Spadaro; C De Carolis; M Cancian Journal: Front Med (Lausanne) Date: 2022-09-14
Authors: Aleena Banerji; Jonathan A Bernstein; Douglas T Johnston; William R Lumry; Markus Magerl; Marcus Maurer; Inmaculada Martinez-Saguer; Andrea Zanichelli; James Hao; Neil Inhaber; Ming Yu; Marc A Riedl Journal: Allergy Date: 2021-08-13 Impact factor: 14.710