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ERJ Open Res. 2019 Feb 1;5(1). pii: 00147-2018. doi: 10.1183/23120541.00147-2018. eCollection 2019 Feb.

Pulmonary exacerbations in patients with primary ciliary dyskinesia: an expert consensus definition for use in clinical trials.

Author information

1
Primary Ciliary Dyskinesia Centre, NIHR Southampton Biomedical Research Centre, University of Southampton and University Hospital Southampton NHS Foundation Trust, Southampton, UK.
2
Pulmonology Dept, Centro Hospitalar S. João, Porto, Portugal.
3
Faculty of Medicine, Porto University, Porto, Portugal.
4
Dept of Paediatrics, University Hospital Gasthuisberg, Leuven, Belgium.
5
PCD Family Support Group, UK.
6
Paediatric Pulmonology Unit, Paediatrics Dept, Centro Hospitalar Lisboa Norte, Lisbon Academic Medical Centre, Lisbon, Portugal.
7
Paediatric Dept of Allergy and Lung Diseases, Oslo University Hospital, Oslo, Norway.
8
Association des Patients Ayant une Dyskinésie Ciliaire Primitive, France.
9
Division of Paediatric Pulmonology and Allergology, Dept of Paediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria.
10
Schneider Children's Medical Center of Israel, Petach-Tikva and Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel.
11
Dept of Pediatric Pulmonology, VU University Medical Center, Amsterdam, The Netherlands.
12
Dept of Paediatric Pneumology, University Children's Hospital of Ruhr University, Bochum, Germany.
13
Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
14
Paediatric Respiratory Medicine, University Children's Hospital of Bern, University of Bern, Bern, Switzerland.
15
Host Defence Unit, Royal Brompton and Harefield NHS Foundation Trust, London, UK.
16
Imperial College London, London, UK.
17
Dept of Respiratory Medicine, University Hospitals Leuven, Leuven, Belgium.
18
Service de Pneumologie, Hôpital Intercommunal de Créteil, DHU A-TVB, Inserm U955, Université Paris Est-Créteil, Créteil, France.
19
Section of Pediatric Allergy, Pulmonology and Cystic Fibrosis, Hospital Vall d'Hebron, Universitat Autónoma de Barcelona, CIBERER, ISCIII, Barcelona, Spain.
20
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.
21
Dept of Pediatric Pulmonology, University Medical Faculty, Ankara, Turkey.
22
Paediatric Dept, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic.
23
Vall d'Hebron Research Institute, Respiratory Disease Dept, Hospital Vall d'Hebron, CIBER, Barcelona, Spain.
24
Dept of Respiratory Medicine, Hannover Medical School, Hanover, Germany.
25
PCD Service, Dept of Respiratory and Sleep Medicine, Royal Children's Hospital, Melbourne, Australia.
26
Primary Ciliary Dyskinesia Centre, Dept of Woman and Child Health (SDB), University of Padova, Padua, Italy.
27
Medical School, University of Cyprus, Nicosia, Cyprus.
28
Primary Ciliary Dyskinesia Centre, Dept of Paediatric Respiratory Medicine, Imperial College and Royal Brompton Hospital, London, UK.

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.

Conflict of interest statement

Conflict of interest: J.S. Lucas reports grants, personal fees and nonfinancial support from Aerocrine/Circassia, grants and personal fees from Vertex, and grants from Parion, outside the submitted work. Conflict of interest: F. Gahleitner has nothing to disclose. Conflict of interest: A. Amorim reports personal fees from Zambon and personal fees from Vertex, outside the submitted work. Conflict of interest: M. Boon reports a postdoctoral research grant from KOOR, University Hospital Leuven, outside the submitted work. Conflict of interest: P. Brown has nothing to disclose. Conflict of interest: C. Constant has nothing to disclose. Conflict of interest: S. Cook has nothing to disclose. Conflict of interest: S. Crowley has nothing to disclose. Conflict of interest: D.M.S. Destouches has nothing to disclose. Conflict of interest: E. Eber has nothing to disclose. Conflict of interest: H. Mussaffi has nothing to disclose. Conflict of interest: E. Haarman has nothing to disclose. Conflict of interest: A. Harris has nothing to disclose. Conflict of interest: C. Koerner-Rettberg has nothing to disclose. Conflict of interest: C.E. Kuehni has nothing to disclose. Conflict of interest: P. Latzin reports personal fees from Vertex, Novartis, Roche, Polyphor, Vifor, Gilead, Schwabe, Zambon, Santhera and Vertex, outside the submitted work. Conflict of interest: M.R. Loebinger reports personal fees from Bayer, Griffols, Polyphor and Raptor, and other support from Parion, outside the submitted work. Conflict of interest: N. Lorent reports personal fees from Chiesi, and nonfinancial support from Insmed and Mylan, outside the submitted work. Conflict of interest: B. Maitre has nothing to disclose. Conflict of interest: A. Moreno-Galdó reports personal fees from AbbVie, and other support from AbbVie, Actelion and Novartis, outside the submitted work. Conflict of interest: K.G. Nielsen has nothing to disclose. Conflict of interest: U. Özçelik has nothing to disclosure Conflict of interest: L.K.D. Philipsen has nothing to disclose. Conflict of interest: P. Pohunek reports personal fees from Novartis, Sandoz and Teva, grants from Teva, and personal fees from Stallergenes, S&D Pharma, ALK and Boehringer Ingelheim, outside the submitted work. Conflict of interest: E. Polverino reports personal fees from Bayer, Insmed and Grifols, Zambon, grants and personal fees from Chiesi, and personal fees from Polyphor, during the conduct of the study; none of these fees and grants were strictly related with the topic of this manuscript. Conflict of interest: J. Rademacher has nothing to disclose. Conflict of interest: P. Robinson has nothing to disclose. Conflict of interest: D. Snijders has nothing to disclose. Conflict of interest: P. Yiallouros has nothing to disclose. Conflict of interest: S.B. Carr reports personal fees and other support from Vertex Pharmaceuticals, other support from Chiesi Ltd, personal fees from Teva Pharmaceutical Industries and other support from Pharmaxis Pharmaceuticals Ltd, outside the submitted work.

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