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

Y1 - 2024

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.

U2 - 10.1183/23120541.00115-2023

DO - 10.1183/23120541.00115-2023

M3 - Journal article

C2 - 38196895

AN - SCOPUS:85182864524

VL - 10

JO - ERJ Open Research

JF - ERJ Open Research

SN - 2312-0541

IS - 1

M1 - 00115-2023

ER -