Bronchiectasis is a clinical syndrome of recurrent or persistent wet/productive cough, airway infection and inflammation, with objectively documented abnormal bronchial dilatation on chest computed-tomography (CT) scans.
As children differ from adults in many ways including in physiological, developmental, psychological and pharmacological characteristics, there is an increasing appreciation of the need for paediatric-based trials instead of relying on the extrapolation of adult-data. Thus while an adult COS exist https://www.comet-initiative.org/Studies/Details/936, one specific for children are required to improve the impact of clinical research.
We aim to develop a consensus on a set of well-defined patient-relevant outcomes/end-points for clinical interventions that will inform the planning and conduct of future studies to improve the relevance of future studies related to paediatric bronchiectasis.
To develop a core outcome set for clinical trials in children and adolescents with bronchiectasis
ContributorsAnne B Chang1,2, Jeanette Boyd3, Andrew Bush4, Adam T Hill5, Zena Powell6, Angela Zacharasiewicz7, Efthymia Alexopoulou8, Andrew J Collaro1, James D Chalmers9, Carolina Constant10, Kostas Douros11, Rebecca Fortescue12, Matthias Griese13, Jonathan Grigg14, Andreas Hector15, Bulent Karadag16, Oleksandr Mazulov17, Fabio Midulla18, Alexander Möller19, Marijke Proesmans20, Christine Wilson21, Stephanie T Yerkovich1,2, *Keith Grimwood22, *Ahmad Kantar23
1Australian Centre for Health Services Innovation, Queensland University of Technology, Brisbane, Queensland and Department of Respiratory and Sleep Medicine, Queensland Children’s Hospital, Australia;
2NHMRC Centre for Research Excellence in Paediatric Bronchiectasis (AusBREATHE), Child Health Division, Menzies School of Health Research, Charles Darwin University, Australia;
3European Lung Foundation, Sheffield, United Kingdom;
4Department of Paediatric Respiratory Medicine, Royal Brompton Hospital, and National Heart and Lung Institute, Imperial School of Medicine, London, UK;
5Dept of Respiratory Medicine, Royal Infirmary and University of Edinburgh, Edinburgh, UK;
6European Lung Foundation bronchiectasis paediatric patient advisory group;
7Department of Pediatrics, and Adolescent Medicine, Teaching Hospital of the University of Vienna, Wilhelminen Hospital, Klinikum Ottakring Vienna, Austria;
82nd Radiology Department, National and Kapodistrian University of Athens, Attikon University Hospital, Athens, Greece;
9College of Medicine, University of Dundee, Ninewells Hospital and Medical School, Dundee, UK;
10Department of Pediatrics, Hospital de Santa Maria and Faculty of Medicine, University of Lisbon, Lisbon, Portugal;
11Allergology and Pulmonology Unit, 3rd Paediatric Dept., National and Kapodistrian University of Athens, Greece;
12Population Health Research Institute, St George’s University of London, United Kingdom;
13Department of Pediatrics, Dr. von Hauner Children's Hospital, University Hospital, Ludwig-Maximilians-Universität Munich, Munich, Germany;
14Centre for Genomics and Child Health, Blizard Institute, Queen Mary University of London, London, UK;
15Department of Pediatrics, University Hospital Tuebingen, Hoppe-Seyler-Strasse 1, 72076, Tuebingen, Germany;
16Division of Pediatric Pulmonology, Marmara University Faculty of Medicine, Istanbul, Turkey;
17National Pirogov Medical University, Vinnytsya Children's Regional Hospital, Pulmonology Dept, Vinnytsya, Ukraine;
18Department of Maternal Science, Sapienza University of Rome;
19Division of Respiratory Medicine, University Children’s Hospital Zuerich and Childhood Research Center, Zuerich, Switzerland;
20Department of Pediatrics, Pediatric Pulmonology, University Hospital of Leuven, Leuven, Flanders, Belgium
21Department of Physiotherapy, Queensland Children’s Hospital, Brisbane, Queensland;
22NHMRC Centre for Research Excellence in Paediatric Bronchiectasis (AusBREATHE), Departments of Infectious Disease and Paediatrics, Gold Coast Health, Southport, Queensland, Australia; School of Medicine and Dentistry, Menzies Health Institute Queensland, Griffith University, Gold Coast campus, Southport, Queensland, Australia;
23Pediatric Asthma and Cough Centre, Istituti Ospedalieri Bergamaschi, University and Research Hospitals, via Forlanini 15, Ponte San Pietro-Bergamo, 24036, Bergamo, Italy.
PI=Anne Chang
Disease Category: Lungs & airways
Disease Name: Paediatric bronchiectasis
Age Range: 0 - 18
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Consumers (caregivers)
- Consumers (patients)
- Epidemiologists
- Families
- Researchers
- Service providers
- Service users
- Statisticians
- COS for clinical trials or clinical research
- Consensus meeting
- Focus group(s)
- Semi structured discussion
- Systematic review
1. Systematic review of current outcomes and outcome measurement instruments in interventions for bronchiectasis studies.
2. Focus meeting with panel of experts and parents of children with bronchiectasis patients to identify additional important outcomes from stakeholders’ point of view.
3. Pilot survey for ranking of outcomes
4. Survey monkey sent out internationally to health professionals and parents/patients
5. Discuss results with parent advisory group
6. Consensus meeting to agree final COS based on the results of the survey and feedback from parent advisory group