A core outcome set for efficacy of hereditary angioedema treatment
Published: April 10, 2024
Hereditary angioedema is a rare genetic disorder that manifests with unpredictable swellings that can be debilitating, extremely painful, and sometimes fatal. Clinical trials investigating drugs to treat these swellings have used many different outcomes to assess efficacy. This difference in measured outcomes hinders comparison between trials, and the measuring of numerous outcomes within one trial imposes a burden on trial participants and may risk selective outcome reporting bias. The AcUte tReatment Outcomes in heReditary Angioedema (AURORA) project aimed to develop a core outcome set consisting of key outcomes that should be measured and reported in all clinical trials of acute treatment of hereditary angioedema swellings.
Petersen et al. report in The Journal of Allergy and Clinical Immunology: In Practice the conduct of a Delphi process, a consensus-building method that involves the use of successive anonymous survey rounds to gather input from a selected expert panel. In AURORA, all relevant stakeholders were invited, including clinicians, patients, Industry representatives and regulatory agencies. A consensus report was developed based on the results of two survey rounds, which was approved for submission by all authors.
A high level of consensus was achieved on a core outcome set for measuring efficacy of acute treatment of hereditary angioedema attacks, among 58 participants from 23 countries who completed both survey rounds. The participants agreed on the following five critical outcomes:
1. Change in overall symptom severity at a predetermined time point between 15 minutes and 4 hours after treatment
2. Time to end of progression of all angioedema symptoms
3. Need for rescue medication
4. Impairment of daily activities
5. Treatment satisfaction
This core outcome set should be applied in all future clinical trials investigating acute treatment for hereditary angioedema.
The Journal of Allergy and Clinical Immunology: In Practice is an official journal of the AAAAI, focusing on practical information for the practicing clinician.
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