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

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Chang, Anne B
Boyd, Jeanette
Bush, Andrew
Hill, Adam T
Powell, Zena
Zacharasiewicz, Angela
Alexopoulou, Efthymia
Collaro, Andrew J
Chalmers, James D
Constant, Carolina
Douros, Konstantinos
Fortescue, Rebecca
Griese, Matthias
Grigg, Jonathan
Hector, Andreas
et al.
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2023
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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.

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The Lancet Respiratory Medicine

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This publication has been entered in Griffith Research Online as an advanced online version.

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Cardiovascular medicine and haematology

Clinical sciences

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Chang, AB; Boyd, J; Bush, A; Hill, AT; Powell, Z; Zacharasiewicz, A; Alexopoulou, E; Collaro, AJ; Chalmers, JD; Constant, C; Douros, K; Fortescue, R; Griese, M; Grigg, J; Hector, A; et al., A core outcome set for bronchiectasis in children and adolescents for use in clinical research: an international consensus study, The Lancet Respiratory Medicine, 2023

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