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.
Original language | English |
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Pages (from-to) | 78-88 |
Number of pages | 11 |
Journal | The Lancet Respiratory Medicine |
Volume | 12 |
Issue number | 1 |
DOIs | |
Publication status | Published - Jan 2024 |
Bibliographical note
Funding Information:This study was partially funded by the European Respiratory Society (ERS) Children's Bronchiectasis Education, Advocacy and Research Network (Child-BEAR-Net). Child-BEAR-Net is funded by an ERS Clinical Research Collaboration grant. ABC and KG report grants from the Australian National Health and Medical Research Council (NHMRC) and the Medical Research Futures Fund (NHMRC-managed grants) during the conduct of the study. ABC is also supported by a NHMRC senior practitioner fellowship (APP1154302). AJC is supported by a NHMRC postgraduate scholarship (APP2003334). The funding sources had no role in the study design, data collection, data analysis, data interpretation, writing of the report, or the decision to submit the paper for publication. We thank Elizabeth Stovold, Information Specialist from the Cochrane Airways Group, for designing and undertaking the literature searches. We are grateful to the patient advisory groups of the European Lung Foundation and AusBREATHE for their reflections and advice during survey development, the pilot phase, and consensus process. We also thank all the people who responded to the survey. We appreciate the support for Child-BEAR-Net of Elise Heuvelin, Celine Genton, and Joanna Pepke-Zaba from the ERS Clinical Research Collaboration office.
Funding Information:
This study was partially funded by the European Respiratory Society (ERS) Children's Bronchiectasis Education, Advocacy and Research Network (Child-BEAR-Net). Child-BEAR-Net is funded by an ERS Clinical Research Collaboration grant. ABC and KG report grants from the Australian National Health and Medical Research Council (NHMRC) and the Medical Research Futures Fund (NHMRC-managed grants) during the conduct of the study. ABC is also supported by a NHMRC senior practitioner fellowship (APP1154302). AJC is supported by a NHMRC postgraduate scholarship (APP2003334). The funding sources had no role in the study design, data collection, data analysis, data interpretation, writing of the report, or the decision to submit the paper for publication. We thank Elizabeth Stovold, Information Specialist from the Cochrane Airways Group, for designing and undertaking the literature searches. We are grateful to the patient advisory groups of the European Lung Foundation and AusBREATHE for their reflections and advice during survey development, the pilot phase, and consensus process. We also thank all the people who responded to the survey. We appreciate the support for Child-BEAR-Net of Elise Heuvelin, Celine Genton, and Joanna Pepke-Zaba from the ERS Clinical Research Collaboration office.
Publisher Copyright:
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