A BEAT-PCD consensus statement: a core outcome set for pulmonary disease interventions in primary ciliary dyskinesia

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

• Kos, R.
• Goutaki, M.
• Kobbernagel, H. E.
• Rubbo, B.
• Shoemark, A.
• Aliberti, S.
• Altenburg, J.
• Anagnostopoulou, P.
• Athanazio, R. A.
• Beydon, N.
• Dell, S. D.
• Emiralioglu, N.
• Ferkol, T. W.
• Loebinger, M. R.
• Lorent, N.
• Maître, B.
• Marthin, J.
• Morgan, L. C.
• Nielsen, K. G.
• Ringshausen, F. C.
• Shteinberg, M.
• Tiddens, Hawm
• Maitland-Van der Zee, A. H.
• Chalmers, J. D.
• Lucas, J. S. A.
• Haarman, E. G.