Tables
- TABLE 1
Clinical trials in primary ciliary dyskinesia (PCD) patients that used a definition for pulmonary exacerbations
First author [ref.]# Study aims Study population Method used to develop the definition Definition of exacerbation Kobbernagel [16] Protocol for randomised controlled trial to determine the efficacy and safety of azithromycin maintenance therapy PCD children aged >7 years and adults Face-to-face discussion at BESTCILIA study meeting Either Respiratory symptoms (not listed) leading to start of systemic antibiotic treatment, irrespective of results of bacterial culture or Decline in FEV1 % pred ≥10% relative to the FEV1 % pred at randomisation, irrespective of whether antibiotics are prescribed Ratjen [17] Changes in airway inflammation during pulmonary exacerbations Cystic fibrosis and PCD children aged >6 years Researcher defined Increase in respiratory symptoms (not listed) treated with oral antibiotics Sunther [15] Recovery of baseline lung function after pulmonary exacerbation PCD children Researcher defined A change in respiratory status for which intravenous antibiotics were prescribed FEV1: forced expiratory volume in 1 s. #: all three trials were published in 2016.
- TABLE 2
Results of voting for which criteria should contribute to the definition
Potential criteria for inclusion Survey 2# Survey 3¶
calculated ranking
(1=most important)Survey 4+
% agreementMean score % agree Change in sputum volume and/or colour 1.54 93 2 96 Increased cough 1.64 89 1 100 New/increased haemoptysis 1.86 79 8 92 Increased shortness of breath (parent/patient perceived) 1.54 100 3 92 Increased respiratory rate 2.14 64 Increased chest discomfort/chest pain 2.07 75 Malaise, tiredness, fatigue or lethargy 2.14 68 7 92 Decreased activity 2.54 NA Decreased exercise tolerance 2.14 71 Temperature >38°C 1.96 82 9 84 Anorexia or weight loss 2.50 NA Change in physical examination of the chest 2.29 NA Increased crepitations/crackles 2.21 68 Increased wheeze 2.43 NA New radiographic changes indicative of a pulmonary infection 2.18 75 5 Decrease in pulmonary function of ≥10% from a previously recorded value (FEV1 % pred or FVC % pred) 2.11 75 4 Raised C-reactive protein 2.79 NA Prolonged erythrocyte sedimentation rate 3.39 NA Raised white cell count 3.00 NA Raised neutrophil count 2.96 NA Physician decision to change treatment because of perceived change in condition 6 92 FEV1: forced expiratory volume in 1 s; FVC: forced vital capacity; NA: neither agree nor disagree. #: in Survey 2, participants (n=28) indicated whether each item should be included in the definition of pulmonary exacerbations (1=strongly agree; 5=strongly disagree). The mean score and the percentage of respondents who agreed are shown. ¶: items which were considered positively in Survey 2 were ranked in Survey 3 (n=25 participants); “physician decision to treat” was added to the list for ranking. We present the calculated rank score from all participants. +: in Survey 4 (n=25 participants), the final wording for included items was agreed. The items included in the final definition are in italics.
- TABLE 3
Definition of a pulmonary exacerbation for children and adults with primary ciliary dyskinesia (PCD) participating in clinical research
The following definition can be used in clinical research to define a pulmonary exacerbation in children and adults with PCD: Three or more of the following must be present: Increased cough
Change in sputum volume and/or colour
Increased shortness of breath perceived by the patient or parent
Decision to start or change antibiotic treatment because of perceived pulmonary symptoms
Malaise, tiredness, fatigue or lethargy
New or increased haemoptysis
Temperature >38°C
Supplementary Materials
Supplementary Material
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Pulmonary Exacerbations Consensus Survey 00147-2018_supp
Supplementary tables 00147-2018_supp_tables
