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
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.
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