A core outcome set for bronchiectasis in children and adolescents for use in clinical research: an international consensus study

Abstract

Improving the treatment of non-cystic fibrosis bronchiectasis in children and adolescents requires high-quality research with outcomes that meet study objectives and are meaningful for patients and their parents and caregivers. In the absence of systematic reviews or agreement on the health outcomes that should be measured in paediatric bronchiectasis, we established an international, multidisciplinary panel of experts to develop a core outcome set (COS) that incorporates patient and parent perspectives. We undertook a systematic review from which a list of 21 outcomes was constructed; these outcomes were used to inform the development of separate surveys for ranking by parents and patients and by health-care professionals. 562 participants (201 parents and patients from 17 countries, 361 health-care professionals from 58 countries) completed the surveys. Following two consensus meetings, agreement was reached on a ten-item COS with five outcomes that were deemed to be essential: quality of life, symptoms, exacerbation frequency, non-scheduled health-care visits, and hospitalisations. Use of this international consensus-based COS will ensure that studies have consistent, patient-focused outcomes, facilitating research worldwide and, in turn, the development of evidence-based guidelines for improved clinical care and outcomes. Further research is needed to develop validated, accessible measurement instruments for several of the outcomes in this COS.; Competing Interests: Declaration of interests ABC is a member of the independent data management committees for clinical trials for Moderna (COVID-19 vaccine), GSK (an unlicensed vaccine), and AstraZeneca (a monoclonal antibody); she also reports payments to her institution for consulting on study designs for Zambon and Boehringer Ingelheim, travel expenses from the European Respiratory Society and Boehringer Ingelheim, and personal fees for authorship of two UpToDate chapters, outside of the submitted work. AZ reports personal fees for lectures from AstraZeneca, Chiesi, Vertex Pharmaceuticals, and Sanofi, and travel fees from Vertex, outside of the submitted work. JDC reports personal consulting fees from AstraZeneca, Boehringer Ingelheim, Chiesi, GSK, Insmed, Grifols, Pfizer, Jansen, Antabio, and Zambon, outside of the submitted work but related to bronchiectasis in adults; he also reports grants from AstraZeneca, Boehringer Ingelheim, Novartis, GSK, Gilead Sciences, Insmed, Grifols, and Genentech, outside of the submitted work but related to bronchiectasis in adults. MG reports personal consulting fees and honoraria for lectures and presentations and for advice on an adjudication board from Boehringer Ingelheim and for advice on study development from Roche, outside of the submitted work. JG reports unrestricted grants from OM Pharma and Mariomed Biotech, and receipt of wheeze-detection equipment without cost from OMRON; he also reports personal fees for advisory board membership from OM Pharma, GSK, and AstraZeneca, for his role as a chief investigator on an asthma study from AstraZeneca, for lectures from Sanofi, and for expert testimony for medical advice, outside of the submitted work. AM reports grants to his institution from Vertex. All other authors declare no competing interests. (Copyright © 2023 Elsevier Ltd. All rights reserved.)