A Core Outcome Set for Efficacy of Acute Treatment of Hereditary Angioedema

Petersen R.S. Fijen L.M. Apfelbacher C. Magerl M. Weller K. Aberer W. Adatia A. Audhya P. Bara N.-A. Betschel S. Boccon-Gibod I. Bouillet L. Brodszki N. Busse P.J. Buttgereit T. Bygum A. Cancian M. Craig T. Csuka D. Farkas H. Fomina D. Gil-Serrano J. Gompels M. Guidos Fogelbach G. Guilarte M. Hide M. Kiani-Alikhan S. Kinaciyan T. Lenten A. lleonart R. Longhurst H. Lumry W.R. Malbran A. Malinauskiene L. Matta Campos J.J. Mendivil J. Nieto-Martinez S.A. Peter J.G. Porebski G. Reshef A. Riedl M. Valerieva A. Waserman S. Maurer M. Cohn D.M.
June 2024 American Academy of Allergy, Asthma and Immunology

Journal of Allergy and Clinical Immunology: In Practice
2024 #12 Issue 6 1614 - 1621 pp.

Background: Clinical trials investigating drugs for the acute treatment of hereditary angioedema attacks have assessed many different outcomes. This heterogeneity limits the comparability of trial results and may lead to selective outcome reporting bias and a high burden on trial participants. Objective: To achieve consensus on a core outcome set composed of key outcomes that ideally should be used in all clinical efficacy trials involving the acute treatment of hereditary angioedema attacks. Methods: We conducted a Delphi consensus study involving all relevant parties: patients with hereditary angioedema, hereditary angioedema expert clinicians and clinical researchers, pharmaceutical companies, and regulatory bodies. Two Internet-based survey rounds were conducted. In round 1, panelists indicated the importance of individual outcomes used in clinical trials on a 9-point Likert scale. Based on these results, a core outcome set was developed and voted on by panelists in round 2. Results: A total of 58 worldwide panelists completed both rounds. The first round demonstrated high importance scores and substantial agreement among the panelists. In the second round, a consensus of 90% or greater was achieved on a core outcome set consisting of five key outcomes: change in overall symptom severity at one predetermined time point between 15 minutes and 4 hours after treatment, time to end of progression of all symptoms, the need for rescue medication during the entire attack, impairment of daily activities, and treatment satisfaction. Conclusions: This international study obtained a high level of consensus on a core outcome set for the acute treatment of hereditary angioedema attacks, consisting of five key outcomes.

Acute treatment , Consensus , Core outcome set , Delphi , Hereditary angioedema , Outcome , Randomized controlled trial

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Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, Amsterdam, Netherlands
Institute of Social Medicine and Health Systems Research, Medical Faculty, Otto-von-Guericke University Magdeburg, Magdeburg, Germany
Angioedema Center of Reference and Excellence, Institute of Allergology, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany
Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Immunology and Allergology, Berlin, Germany
Department of Dermatology, Medical University of Graz, Graz, Austria
Division of Pulmonary Medicine, Department of Medicine, University of Alberta, Edmonton, AB, Canada
KalVista Pharmaceuticals, Cambridge, Mass, United States
Romanian Hereditary Angioedema Expertise Centre, Mediquest Clinical Research Center, Sangeorgiu de Mures, Romania
Department of Medicine, St Michaels Hospital, University of Toronto, Toronto, ON, Canada
National Reference Center for Angioedema (CREAK), Angioedema Center of Reference and Excellence, CHU Grenoble Alpes, France
University of Grenoble Alpes, CNRS, UMR 5525, VetAgro Sup, Grenoble INP, CHU Grenoble Alpes, TIMC, Grenoble, France
Department of Pediatric Immunology, Childrens Hospital, Skåne University Hospital, Lund, Sweden
Division of Allergy and Clinical Immunology, Icahn School of Medicine at Mount Sinai, New York, NY, United States
Clinical Institute, University of Southern Denmark, Odense, Denmark
Department of Systems Medicine, University Hospital of Padua, Padua, Italy
Allergy, Asthma, and Immunology Division, Department of Medicine and Pediatrics, Penn State University, Hershey, Pa, United States
Department of Internal Medicine and Haematology, Hungarian Angioedema Center of Reference and Excellence, Semmelweis University, Budapest, Hungary
Urticaria Center of Reference and Excellence, Moscow Research and Clinical Center of Allergy and Immunology, Clinical City Hospital 52, Moscow, Russian Federation
Department of Clinical Immunology and Allergology, Sechenov University, Moscow, Russian Federation
Department of Pulmonology, Astana Medical University, Astana, Kazakhstan
Allergy Section Department, Department of Internal Medicine, Hospital Universitari Vall d’ Hebron, Barcelona, Spain
Allergy Research Unit, Allergy Department, Institut de Recerca Vall d’ Hebron, Universitat Autònoma de Barcelona, Spain
Clinical Immunology, North Bristol NHS Trust, Bristol, United Kingdom
Department of Immunology, Instituto Politécnico Nacional SEPI-ENMH, Mexico City, Mexico
Department of Dermatology, Hiroshima City Hiroshima Citizens Hospital, Hiroshima, Japan
Royal Free London NHS Foundation Trust, London, United Kingdom
Department of Dermatology, Medical University of Vienna, Vienna, Austria
Takeda Pharmaceuticals, Hoofddorp, Netherlands
Allergology Department, Hospital Universitari de Bellvitge, Institut de Recerca IDIBELL LHospitalet de Llobregat, Barcelona, Spain
Department of Immunology, Auckland District Health Board and Department of Medicine, University of Auckland, Auckland, New Zealand
Internal Medicine, Allergy Division, University of Texas Health Science Center, Dallas, Tex, United States
Unidad de Alergia, Asma e Inmunologia Clinica, Buenos Aires, Argentina
Medical Faculty, Clinic of Chest Diseases, Immunology and Allergology, VUH Santaros Klinikos, Department of Pulmonology and Allergology, Vilnius University, Vilnius, Lithuania
Allergy and Clinical Immunology Department, UMAE Hospital Especialidades CMNSXXI, IMSS, México City, Mexico
Head Evidence and Outcomes Research, Pharvaris GmbH, Zug, Switzerland
Genetic Unit of Nutrition, National Institute of Pediatrics, México City, Mexico
Division of Allergy and Clinical Immunology, Groote Schuur Hospital, University of Cape Town and Allergy and Immunology Unit, University of Cape Town Lung Institute, Cape Town, South Africa
Department of Clinical and Environmental Allergology, Jagiellonian University Medical College, Krakow, Poland
Angioedema Center, Barzilai University Medical Center, Ashkelon, Israel
Division of Rheumatology, Allergy, and Immunology, University of California San Diego, La Jolla, Calif, United States
Department of Allergology, Medical University of Sofia and Angioedema Center of Reference and Excellence Bulgaria (University Hospital Alexandrovska), Sofia, Bulgaria
Department of Medicine, McMaster University, Hamilton, ON, Canada

Department of Vascular Medicine
Institute of Social Medicine and Health Systems Research
Angioedema Center of Reference and Excellence
Fraunhofer Institute for Translational Medicine and Pharmacology ITMP
Department of Dermatology
Division of Pulmonary Medicine
KalVista Pharmaceuticals
Romanian Hereditary Angioedema Expertise Centre
Department of Medicine
National Reference Center for Angioedema (CREAK)
University of Grenoble Alpes
Department of Pediatric Immunology
Division of Allergy and Clinical Immunology
Clinical Institute
Department of Systems Medicine
Allergy
Department of Internal Medicine and Haematology
Urticaria Center of Reference and Excellence
Department of Clinical Immunology and Allergology
Department of Pulmonology
Allergy Section Department
Allergy Research Unit
Clinical Immunology
Department of Immunology
Department of Dermatology
Royal Free London NHS Foundation Trust
Department of Dermatology
Takeda Pharmaceuticals
Allergology Department
Department of Immunology
Internal Medicine
Unidad de Alergia
Medical Faculty
Allergy and Clinical Immunology Department
Head Evidence and Outcomes Research
Genetic Unit of Nutrition
Division of Allergy and Clinical Immunology
Department of Clinical and Environmental Allergology
Angioedema Center
Division of Rheumatology
Department of Allergology
Department of Medicine

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