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

Mar Guilarte, Stephen Betschel, Isabelle Boccon-Gibod, Laurence Bouillet, Nicholas Brodszki, Paula J Busse, Thomas Buttgereit, Mauro Cancian, Dorottya Csuka, Daria Fomina, Johana Gil-Serrano, Guillermo Guidos Fogelbach, Noémi-Anna Bara, Annet Lenten, Ramon Lleonart, Alejandro Malbran, Laura Malinauskiene, Juan J Matta Campos, Joan Mendivil, Jonathan G Peter, Grzegorz Porebski, Avner Reshef, Anna Valerieva, Marc Riedl, Lauré M Fijen, Danny M Cohn, Sandra A Nieto-Martinez, Sorena Kiani-Alikhan, Hilary Longhurst, Mark Gompels, Henriette Farkas, Paul Audhya, William R Lumry, Tamar Kinaciyan, Michihiro Hide, Susan Waserman, Timothy Craig, Karsten Weller, Markus Magerl, Anette Bygum, Marcus Maurer, Christian Apfelbacher, Remy S Petersen, Werner Aberer, Adil Adatia

Journal: The journal of allergy and clinical immunology. In practice 2024;12(6):1614-1621

PMID: 38609017

Abstract

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.

Crown Copyright © 2024. Published by Elsevier Inc. All rights reserved.

Address: Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, Amsterdam, the 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, Alberta, Canada.; KalVista Pharmaceuticals, Cambridge, Mass.; Romanian Hereditary Angioedema Expertise Centre, Mediquest Clinical Research Center, Sangeorgiu de Mures, Romania.; Department of Medicine, St Michael's Hospital, University of Toronto, Toronto, Ontario, Canada.; National Reference Center for Angioedema (CREAK), Angioedema Center of Reference and Excellence, CHU Grenoble Alpes, France.; 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.; 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.; 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, Russia; Department of Clinical Immunology and Allergology, Sechenov University, Moscow, Russia; 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, the Netherlands.; Allergology Department, Hospital Universitari de Bellvitge, Institut de Recerca IDIBELL L'Hospitalet 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.; 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.; 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, Ontario, Canada.; Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands. Electronic address: [email protected].
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