TY - JOUR
T1 - A BEAT-PCD consensus statement
T2 - a core outcome set for pulmonary disease interventions in primary ciliary dyskinesia
AU - Kos, Renate
AU - Goutaki, Myrofora
AU - Kobbernage, Helene E.
AU - Rubbo, Bruna
AU - Shoemark, Amelia
AU - Aliberti, Stefano
AU - Altenburg, Josje
AU - Anagnostopoulou, Pinelopi
AU - Athanazio, Rodrigo A.
AU - Beydon, Nicole
AU - Dell, Sharon D.
AU - Emiralioglu, Nagehan
AU - Ferkol, Thomas W.
AU - Loebinger, Michael R.
AU - Lorent, Natalie
AU - Maître, Bernard
AU - Marthin, June
AU - Morgan, Lucy C.
AU - Nielsen, Kim G.
AU - Ringshausen, Felix C.
AU - Shteinberg, Michal
AU - Tiddens, Harm A. W. M.
AU - Maitland-van der Zee, Anke H.
AU - Chalmers, James D.
AU - Lucas, Jane S. A.
AU - Haarman, Eric G.
N1 - Publisher Copyright: © The authors 2024.
PY - 2024/1/1
Y1 - 2024/1/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85182864524&partnerID=8YFLogxK
U2 - https://doi.org/10.1183/23120541.00115-2023
DO - https://doi.org/10.1183/23120541.00115-2023
M3 - Article
C2 - 38196895
SN - 2312-0541
VL - 10
JO - ERJ open research
JF - ERJ open research
IS - 1
M1 - 00115-2023
ER -