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

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


Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review


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.

TidsskriftThe journal of allergy and clinical immunology. In practice
Udgave nummer6
Sider (fra-til)1614-1621
StatusUdgivet - jun. 2024


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