ERJ Open Research (Feb 2019)
Pulmonary exacerbations in patients with primary ciliary dyskinesia: an expert consensus definition for use in clinical trials
- Jane S. Lucas,
- Florian Gahleitner,
- Adelina Amorim,
- Mieke Boon,
- Philippa Brown,
- Carolina Constant,
- Simon Cook,
- Suzanne Crowley,
- Damien M.S. Destouches,
- Ernst Eber,
- Huda Mussaffi,
- Eric Haarman,
- Amanda Harris,
- Cordula Koerner-Rettberg,
- Claudia E. Kuehni,
- Philipp Latzin,
- Michael R. Loebinger,
- Natalie Lorent,
- Bernard Maitre,
- Antonio Moreno-Galdó,
- Kim G. Nielsen,
- Uğur Özçelik,
- Lue Katrine Drasbæk Philipsen,
- Petr Pohunek,
- Eva Polverino,
- Jessica Rademacher,
- Phil Robinson,
- Deborah Snijders,
- Panayiotis Yiallouros,
- Siobhán B. Carr
Affiliations
- Jane S. Lucas
- Primary Ciliary Dyskinesia Centre, NIHR Southampton Biomedical Research Centre, University of Southampton and University Hospital Southampton NHS Foundation Trust, Southampton, UK
- Florian Gahleitner
- Primary Ciliary Dyskinesia Centre, NIHR Southampton Biomedical Research Centre, University of Southampton and University Hospital Southampton NHS Foundation Trust, Southampton, UK
- Adelina Amorim
- Pulmonology Dept, Centro Hospitalar S. João, Porto, Portugal
- Mieke Boon
- Dept of Paediatrics, University Hospital Gasthuisberg, Leuven, Belgium
- Philippa Brown
- PCD Family Support Group, UK
- Carolina Constant
- Paediatric Pulmonology Unit, Paediatrics Dept, Centro Hospitalar Lisboa Norte, Lisbon Academic Medical Centre, Lisbon, Portugal
- Simon Cook
- PCD Family Support Group, UK
- Suzanne Crowley
- Paediatric Dept of Allergy and Lung Diseases, Oslo University Hospital, Oslo, Norway
- Damien M.S. Destouches
- Association des Patients Ayant une Dyskinésie Ciliaire Primitive, France
- Ernst Eber
- Division of Paediatric Pulmonology and Allergology, Dept of Paediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria
- Huda Mussaffi
- Schneider Children's Medical Center of Israel, Petach-Tikva and Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel
- Eric Haarman
- Dept of Pediatric Pulmonology, VU University Medical Center, Amsterdam, The Netherlands
- Amanda Harris
- Primary Ciliary Dyskinesia Centre, NIHR Southampton Biomedical Research Centre, University of Southampton and University Hospital Southampton NHS Foundation Trust, Southampton, UK
- Cordula Koerner-Rettberg
- Dept of Paediatric Pneumology, University Children's Hospital of Ruhr University, Bochum, Germany
- Claudia E. Kuehni
- Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland
- Philipp Latzin
- Paediatric Respiratory Medicine, University Children's Hospital of Bern, University of Bern, Bern, Switzerland
- Michael R. Loebinger
- Host Defence Unit, Royal Brompton and Harefield NHS Foundation Trust, London, UK
- Natalie Lorent
- Dept of Respiratory Medicine, University Hospitals Leuven, Leuven, Belgium
- Bernard Maitre
- Service de Pneumologie, Hôpital Intercommunal de Créteil, DHU A-TVB, Inserm U955, Université Paris Est-Créteil, Créteil, France
- Antonio Moreno-Galdó
- Section of Pediatric Allergy, Pulmonology and Cystic Fibrosis, Hospital Vall d'Hebron, Universitat Autónoma de Barcelona, CIBERER, ISCIII, Barcelona, Spain
- Kim G. Nielsen
- Danish PCD & chILD Centre, CF Centre Copenhagen Paediatric Pulmonary Service, ERN Accredited for PCD and CF Health Care, Dept of Paediatrics and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark
- Uğur Özçelik
- Dept of Pediatric Pulmonology, University Medical Faculty, Ankara, Turkey
- Lue Katrine Drasbæk Philipsen
- Danish PCD & chILD Centre, CF Centre Copenhagen Paediatric Pulmonary Service, ERN Accredited for PCD and CF Health Care, Dept of Paediatrics and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark
- Petr Pohunek
- Paediatric Dept, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic
- Eva Polverino
- Vall d'Hebron Research Institute, Respiratory Disease Dept, Hospital Vall d'Hebron, CIBER, Barcelona, Spain
- Jessica Rademacher
- Dept of Respiratory Medicine, Hannover Medical School, Hanover, Germany
- Phil Robinson
- PCD Service, Dept of Respiratory and Sleep Medicine, Royal Children's Hospital, Melbourne, Australia
- Deborah Snijders
- Primary Ciliary Dyskinesia Centre, Dept of Woman and Child Health (SDB), University of Padova, Padua, Italy
- Panayiotis Yiallouros
- Medical School, University of Cyprus, Nicosia, Cyprus
- Siobhán B. Carr
- Primary Ciliary Dyskinesia Centre, Dept of Paediatric Respiratory Medicine, Imperial College and Royal Brompton Hospital, London, UK
- DOI
- https://doi.org/10.1183/23120541.00147-2018
- Journal volume & issue
-
Vol. 5,
no. 1
Abstract
Pulmonary exacerbations are a cause of significant morbidity in patients with primary ciliary dyskinesia (PCD) and are frequently used as an outcome measure in clinical research into chronic lung diseases. So far, there has been no consensus on the definition of pulmonary exacerbations in PCD. 30 multidisciplinary experts and patients developed a consensus definition for children and adults with PCD. Following a systematic review, the panel used a modified Delphi process with a combination of face-to-face meetings and e-surveys to develop a definition that can be used in research settings for children and adults with PCD. A pulmonary exacerbation was defined by the presence of three or more of the following seven items: 1) increased cough, 2) change in sputum volume and/or colour, 3) increased shortness of breath perceived by the patient or parent, 4) decision to start or change antibiotic treatment because of perceived pulmonary symptoms, 5) malaise, tiredness, fatigue or lethargy, 6) new or increased haemoptysis, and 7) temperature >38°C. The consensus panel proposed that the definition should be used for future clinical trials. The definition should be validated and the usability assessed during these studies.
