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

Autor:	Petersen RS; Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands., Fijen LM; Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands., Apfelbacher C; Institute of Social Medicine and Health Systems Research, Medical Faculty, Otto-von-Guericke University Magdeburg, Magdeburg, Germany., Magerl M; 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., Weller K; 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., Aberer W; Department of Dermatology, Medical University of Graz, Graz, Austria., Adatia A; Division of Pulmonary Medicine, Department of Medicine, University of Alberta, Edmonton, Alberta, Canada., Audhya P; KalVista Pharmaceuticals, Cambridge, Mass., Bara NA; Romanian Hereditary Angioedema Expertise Centre, Mediquest Clinical Research Center, Sangeorgiu de Mures, Romania., Betschel S; Department of Medicine, St Michael's Hospital, University of Toronto, Toronto, Ontario, Canada., Boccon-Gibod I; National Reference Center for Angioedema (CREAK), Angioedema Center of Reference and Excellence, CHU Grenoble Alpes, France., Bouillet L; 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., Brodszki N; Department of Pediatric Immunology, Childrens Hospital, Skåne University Hospital, Lund, Sweden., Busse PJ; Division of Allergy and Clinical Immunology, Icahn School of Medicine at Mount Sinai, New York, NY., Buttgereit T; 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., Bygum A; Clinical Institute, University of Southern Denmark, Odense, Denmark., Cancian M; Department of Systems Medicine, University Hospital of Padua, Padua, Italy., Craig T; Allergy, Asthma, and Immunology Division, Department of Medicine and Pediatrics, Penn State University, Hershey, Pa., Csuka D; Department of Internal Medicine and Haematology, Hungarian Angioedema Center of Reference and Excellence, Semmelweis University, Budapest, Hungary., Farkas H; Department of Internal Medicine and Haematology, Hungarian Angioedema Center of Reference and Excellence, Semmelweis University, Budapest, Hungary., Fomina D; 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., Gil-Serrano J; 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., Gompels M; Clinical Immunology, North Bristol NHS Trust, Bristol, United Kingdom., Guidos Fogelbach G; Department of Immunology, Instituto Politécnico Nacional SEPI-ENMH, Mexico City, Mexico., Guilarte M; 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., Hide M; Department of Dermatology, Hiroshima City Hiroshima Citizens Hospital, Hiroshima, Japan., Kiani-Alikhan S; Royal Free London NHS Foundation Trust, London, United Kingdom., Kinaciyan T; Department of Dermatology, Medical University of Vienna, Vienna, Austria., Lenten A; Takeda Pharmaceuticals, Hoofddorp, the Netherlands., Lleonart R; Allergology Department, Hospital Universitari de Bellvitge, Institut de Recerca IDIBELL L'Hospitalet de Llobregat, Barcelona, Spain., Longhurst H; Department of Immunology, Auckland District Health Board and Department of Medicine, University of Auckland, Auckland, New Zealand., Lumry WR; Internal Medicine, Allergy Division, University of Texas Health Science Center, Dallas, Tex., Malbran A; Unidad de Alergia, Asma e Inmunologia Clinica, Buenos Aires, Argentina., Malinauskiene L; Medical Faculty, Clinic of Chest Diseases, Immunology and Allergology, VUH Santaros Klinikos, Department of Pulmonology and Allergology, Vilnius University, Vilnius, Lithuania., Matta Campos JJ; Allergy and Clinical Immunology Department, UMAE Hospital Especialidades CMNSXXI, IMSS, México City, Mexico., Mendivil J; Head Evidence and Outcomes Research, Pharvaris GmbH, Zug, Switzerland., Nieto-Martinez SA; Genetic Unit of Nutrition, National Institute of Pediatrics, México City, Mexico., Peter JG; 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., Porebski G; Department of Clinical and Environmental Allergology, Jagiellonian University Medical College, Krakow, Poland., Reshef A; Angioedema Center, Barzilai University Medical Center, Ashkelon, Israel., Riedl M; Division of Rheumatology, Allergy, and Immunology, University of California San Diego, La Jolla, Calif., Valerieva A; Department of Allergology, Medical University of Sofia and Angioedema Center of Reference and Excellence Bulgaria (University Hospital Alexandrovska), Sofia, Bulgaria., Waserman S; Department of Medicine, McMaster University, Hamilton, Ontario, Canada., Maurer M; 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., Cohn DM; Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands. Electronic address: d.m.cohn@amsterdamumc.nl.
Jazyk:	angličtina
Zdroj:	The journal of allergy and clinical immunology. In practice [J Allergy Clin Immunol Pract] 2024 Jun; Vol. 12 (6), pp. 1614-1621. Date of Electronic Publication: 2024 Apr 11.
DOI:	10.1016/j.jaip.2024.04.007
Abstrakt:	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.)
Databáze:	MEDLINE
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