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

Lucas, Jane S; Gahleitner, Florian; Amorim, Adelina; Boon, Mieke; Brown, Philippa; Constant, Carolina; Cook, Simon; Crowley, Suzanne; Destouches, Damien M S; Eber, Ernst; Mussaffi, Huda; Haarman, Eric; Harris, Amanda; Koerner-Rettberg, Cordula; Kuehni, Claudia E; Latzin, Philipp; Loebinger, Michael R; Lorent, Natalie; Maitre, Bernard; Moreno-Galdó, Antonio; ... (2019). Pulmonary exacerbations in patients with primary ciliary dyskinesia: an expert consensus definition for use in clinical trials. ERJ Open Research, 5(1), 00147-2018. European Respiratory Society 10.1183/23120541.00147-2018

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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.

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Paediatric Medicine
04 Faculty of Medicine > Pre-clinic Human Medicine > Institute of Social and Preventive Medicine

UniBE Contributor:

Kühni, Claudia and Latzin, Philipp

Subjects:

300 Social sciences, sociology & anthropology > 360 Social problems & social services
600 Technology > 610 Medicine & health

ISSN:

2312-0541

Publisher:

European Respiratory Society

Language:

English

Submitter:

Beatrice Minder Wyssmann

Date Deposited:

05 Mar 2019 15:07

Last Modified:

23 Oct 2019 23:32

Publisher DOI:

10.1183/23120541.00147-2018

PubMed ID:

30723730

BORIS DOI:

10.7892/boris.127372

URI:

https://boris.unibe.ch/id/eprint/127372

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