1. Pulmonary exacerbations in patients with primary ciliary dyskinesia: an expert consensus definition for use in clinical trials
- Author
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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, Nielsen, Kim G., Özçelik, Uğur, Philipsen, Lue Katrine Drasbæk, Pohunek, Petr, Polverino, Eva, Rademacher, Jessica, Robinson, Phil, Snijders, Deborah, Yiallouros, Panayiotis, Carr, Siobhán B., Universitat Autònoma de Barcelona, Repositório da Universidade de Lisboa, Institut Català de la Salut, [Lucas JS, Gahleitner F] Primary Ciliary Dyskinesia Centre, NIHR Southampton Biomedical Research Centre, University of Southampton and University Hospital Southampton NHS Foundation Trust, Southampton, UK. [Amorim A] Pulmonology Dept, Centro Hospitalar S. João, Porto, Portugal. Faculty of Medicine, Porto University, Porto, Portugal. [Boon M] Dept of Paediatrics, University Hospital Gasthuisberg, Leuven, Belgium. [Brown P] PCD Family Support Group, UK. [Constant C] Paediatric Pulmonology Unit, Paediatrics Dept, Centro Hospitalar Lisboa Norte, Lisbon Academic Medical Centre, Lisbon, Portugal. [Moreno-Galdó A] Servei d'al•lergologia pediàtrica, Hospital Universitari Vall d'Hebron, Barcelona, Spain. Universitat Autònoma de Barcelona, Barcelona, Spain. [Polverino E] Vall d’Hebron Institut de Recerca, Barcelona, Spain. Servei de pneumologia, Hospital Universitari Vall d'Hebron, Barcelona, Spain., Hospital Universitari Vall d'Hebron, Pediatrics, CCA - Cancer biology and immunology, Amsterdam Reproduction & Development (AR&D), Other Research, Vall d'Hebron Barcelona Hospital Campus, Yiallouros, Panayiotis [0000-0002-8339-9285], Latzin, Philipp [0000-0002-5239-1571], and Kuehni, Claudia E. [0000-0001-8957-2002]
- Subjects
Pulmonary and Respiratory Medicine ,medicine.medical_specialty ,Sistema Respiratorio::Pulmón [ANATOMÍA] ,Other subheadings::Other subheadings::/physiopathology [Other subheadings] ,Otros calificadores::Otros calificadores::/fisiopatología [Otros calificadores] ,Primary Ciliary Dyskinesia ,lcsh:Medicine ,610 Medicine & health ,Malaise ,Lethargy ,Enfermedades Respiratorias::Enfermedades Respiratorias::Trastornos de la Motilidad Ciliar::Enfermedades Respiratorias::Síndrome de Kartagener [ENFERMEDADES] ,360 Social problems & social services ,otorhinolaryngologic diseases ,medicine ,In patient ,Intensive care medicine ,Primary ciliary dyskinesia ,Lung ,business.industry ,lcsh:R ,Expert consensus ,Original Articles ,medicine.disease ,Respiratory System::Lung [ANATOMY] ,Clinical trial ,Pulmons - Malalties ,medicine.anatomical_structure ,Clinical research ,Respiratory Tract Diseases::Respiratory Tract Diseases::Ciliary Motility Disorders::Respiratory Tract Diseases::Kartagener Syndrome [DISEASES] ,medicine.symptom ,business - 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., A consensus definition for pulmonary exacerbations in children and adults with PCD for use in clinical trials http://ow.ly/Rcfr30n4Gn4
- Published
- 2019