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A BEAT-PCD consensus statement: a core outcome set for pulmonary disease interventions in primary ciliary dyskinesia.
Kos, Renate; Goutaki, Myrofora; Kobbernagel, Helene E; Rubbo, Bruna; Shoemark, Amelia; Aliberti, Stefano; Altenburg, Josje; Anagnostopoulou, Pinelopi; Athanazio, Rodrigo A; Beydon, Nicole; Dell, Sharon D; Emiralioglu, Nagehan; Ferkol, Thomas W; Loebinger, Michael R; Lorent, Natalie; Maître, Bernard; Marthin, June; Morgan, Lucy C; Nielsen, Kim G; Ringshausen, Felix C; Shteinberg, Michal; Tiddens, Harm A W M; Maitland-Van der Zee, Anke H; Chalmers, James D; Lucas, Jane S A; Haarman, Eric G.
Afiliação
  • Kos R; Dept of Pulmonary Medicine, Amsterdam University Medical Centres - loc. AMC, University of Amsterdam, Amsterdam, The Netherlands.
  • Goutaki M; Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
  • Kobbernagel HE; Paediatric Respiratory Medicine, Children's University Hospital of Bern, University of Bern, Bern, Switzerland.
  • Rubbo B; Danish Primary Ciliary Dyskinesia Centre, Paediatric Pulmonary Service, Dept of Paediatrics and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
  • Shoemark A; Dept of Clinical Medicine, University of Copenhagen, Copenhagen, Denmark.
  • Aliberti S; School of Health Sciences, University of Southampton, Southampton, UK.
  • Altenburg J; Department of Population and Public Health Sciences, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
  • Anagnostopoulou P; Division of Molecular and Clinical Medicine, School of Medicine, University of Dundee, Dundee, UK.
  • Athanazio RA; Dept of Biomedical Sciences, Humanitas University, Milan, Italy.
  • Beydon N; Respiratory Unit, IRCCS Humanitas Research Hospital, Milan, Italy.
  • Dell SD; Dept of Pulmonary Medicine, Amsterdam University Medical Centres - loc. AMC, University of Amsterdam, Amsterdam, The Netherlands.
  • Emiralioglu N; Medical School, University of Cyprus, Nicosia, Cyprus.
  • Ferkol TW; Heart Institute (InCor) Hospital das Clínicas, Faculdade de Medicina da Universidade de São Paulo, Brazil.
  • Loebinger MR; Pulmonary Division, Sorbonne Université, INSERM U938, Paris, France.
  • Lorent N; Unité d'Exploration Fonctionnelle Respiratoire, Hôpital Armand-Trousseau, Paris, France.
  • Maître B; Dept of Paediatrics, Faculty of Medicine, University of British Columbia, Vancouver, Canada.
  • Marthin J; Pediatric Respiratory Medicine, Provincial Health Services Authority, BC Children's Hospital, Vancouver, Canada.
  • Morgan LC; Dept of Pediatric Pulmonology, Hacettepe University Faculty of Medicine, Ankara, Turkey.
  • Nielsen KG; Dept of Pediatrics, University of North Carolina School of Medicine and Marsico Lung Institute, Chapel Hill, NC, USA.
  • Ringshausen FC; Dept of Respiratory Medicine, Royal Brompton and Harefield Hospitals, London, UK.
  • Shteinberg M; National Heart and Lung Institute, Imperial College London, London, UK.
  • Tiddens HAWM; Dept of Pediatrics, University Hospital Leuven, Leuven, Belgium.
  • Maitland-Van der Zee AH; Service de Pneumologie, Hôpital Henri Mondor et Centre Hospitalier Intercommunal de Créteil, Assistance Publique-Hôpitaux de Paris (AP-HP), Créteil, France.
  • Chalmers JD; Danish Primary Ciliary Dyskinesia Centre, Paediatric Pulmonary Service, Dept of Paediatrics and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
  • Lucas JSA; Dept of Microbiology and Infectious Diseases, Concord Repatriation and General Hospital, NSW Health Pathology, Sydney, Australia.
  • Haarman EG; Danish Primary Ciliary Dyskinesia Centre, Paediatric Pulmonary Service, Dept of Paediatrics and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
ERJ Open Res ; 10(1)2024 Jan.
Article em En | MEDLINE | ID: mdl-38196895
ABSTRACT

Background:

Consistent use of reliable and clinically appropriate outcome measures is a priority for clinical trials, with clear definitions to allow comparability. We aimed to develop a core outcome set (COS) for pulmonary disease interventions in primary ciliary dyskinesia (PCD).

Methods:

A multidisciplinary international PCD expert panel was set up. A list of outcomes was created based on published literature. Using a modified three-round e-Delphi technique, the panel was asked to decide on relevant end-points related to pulmonary disease interventions and how they should be reported. First, inclusion of an outcome in the COS was determined. Second, the minimum information that should be reported per outcome. The third round finalised statements. Consensus was defined as ≥80% agreement among experts.

Results:

During the first round, experts reached consensus on four out of 24 outcomes to be included in the COS. Five additional outcomes were discussed in subsequent rounds for their use in different subsettings. Consensus on standardised methods of reporting for the COS was reached. Spirometry, health-related quality-of-life scores, microbiology and exacerbations were included in the final COS.

Conclusion:

This expert consensus resulted in a COS for clinical trials on pulmonary health among people with PCD.

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: ERJ Open Res Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Holanda

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: ERJ Open Res Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Holanda