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Pulmonary exacerbations in patients with primary ciliary dyskinesia: an expert consensus definition for use in clinical trials

Jane S. Lucas, Florian Gahleitner, Adelina Amorim, Mieke Boon, Philippa Brown, Carolina Constant, Simon Cook, Suzanne Crowley, Damien M.S. Destouches, Ernst Eber, Huda Mussaffi, Eric Haarman, Amanda Harris, Cordula Koerner-Rettberg, Claudia E. Kuehni, Philipp Latzin, Michael R. Loebinger, Natalie Lorent, Bernard Maitre, Antonio Moreno-Galdó, Kim G. Nielsen, Uğur Özçelik, Lue Katrine Drasbæk Philipsen, Petr Pohunek, Eva Polverino, Jessica Rademacher, Phil Robinson, Deborah Snijders, Panayiotis Yiallouros, Siobhán B. Carr on behalf of the BEAT-PCD network
ERJ Open Research 2019 5: 00147-2018; DOI: 10.1183/23120541.00147-2018
Jane S. Lucas
1Primary Ciliary Dyskinesia Centre, NIHR Southampton Biomedical Research Centre, University of Southampton and University Hospital Southampton NHS Foundation Trust, Southampton, UK
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  • For correspondence: jlucas1@soton.ac.uk
Florian Gahleitner
1Primary Ciliary Dyskinesia Centre, NIHR Southampton Biomedical Research Centre, University of Southampton and University Hospital Southampton NHS Foundation Trust, Southampton, UK
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Adelina Amorim
2Pulmonology Dept, Centro Hospitalar S. João, Porto, Portugal
3Faculty of Medicine, Porto University, Porto, Portugal
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Mieke Boon
4Dept of Paediatrics, University Hospital Gasthuisberg, Leuven, Belgium
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Philippa Brown
5PCD Family Support Group, UK
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Carolina Constant
6Paediatric Pulmonology Unit, Paediatrics Dept, Centro Hospitalar Lisboa Norte, Lisbon Academic Medical Centre, Lisbon, Portugal
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Simon Cook
5PCD Family Support Group, UK
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Suzanne Crowley
7Paediatric Dept of Allergy and Lung Diseases, Oslo University Hospital, Oslo, Norway
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Damien M.S. Destouches
8Association des Patients Ayant une Dyskinésie Ciliaire Primitive, France
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Ernst Eber
9Division of Paediatric Pulmonology and Allergology, Dept of Paediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria
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Huda Mussaffi
10Schneider Children's Medical Center of Israel, Petach-Tikva and Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel
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Eric Haarman
11Dept of Pediatric Pulmonology, VU University Medical Center, Amsterdam, The Netherlands
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Amanda Harris
1Primary Ciliary Dyskinesia Centre, NIHR Southampton Biomedical Research Centre, University of Southampton and University Hospital Southampton NHS Foundation Trust, Southampton, UK
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Cordula Koerner-Rettberg
12Dept of Paediatric Pneumology, University Children's Hospital of Ruhr University, Bochum, Germany
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Claudia E. Kuehni
13Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland
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  • ORCID record for Claudia E. Kuehni
Philipp Latzin
14Paediatric Respiratory Medicine, University Children's Hospital of Bern, University of Bern, Bern, Switzerland
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Michael R. Loebinger
15Host Defence Unit, Royal Brompton and Harefield NHS Foundation Trust, London, UK
16Imperial College London, London, UK
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Natalie Lorent
17Dept of Respiratory Medicine, University Hospitals Leuven, Leuven, Belgium
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Bernard Maitre
18Service de Pneumologie, Hôpital Intercommunal de Créteil, DHU A-TVB, Inserm U955, Université Paris Est-Créteil, Créteil, France
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Antonio Moreno-Galdó
19Section of Pediatric Allergy, Pulmonology and Cystic Fibrosis, Hospital Vall d'Hebron, Universitat Autónoma de Barcelona, CIBERER, ISCIII, Barcelona, Spain
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Kim G. Nielsen
20Danish PCD & chILD Centre, CF Centre Copenhagen Paediatric Pulmonary Service, ERN Accredited for PCD and CF Health Care, Dept of Paediatrics and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark
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Uğur Özçelik
21Dept of Pediatric Pulmonology, University Medical Faculty, Ankara, Turkey
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Lue Katrine Drasbæk Philipsen
20Danish PCD & chILD Centre, CF Centre Copenhagen Paediatric Pulmonary Service, ERN Accredited for PCD and CF Health Care, Dept of Paediatrics and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark
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Petr Pohunek
22Paediatric Dept, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic
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Eva Polverino
23Vall d'Hebron Research Institute, Respiratory Disease Dept, Hospital Vall d'Hebron, CIBER, Barcelona, Spain
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Jessica Rademacher
24Dept of Respiratory Medicine, Hannover Medical School, Hanover, Germany
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Phil Robinson
25PCD Service, Dept of Respiratory and Sleep Medicine, Royal Children's Hospital, Melbourne, Australia
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Deborah Snijders
26Primary Ciliary Dyskinesia Centre, Dept of Woman and Child Health (SDB), University of Padova, Padua, Italy
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Panayiotis Yiallouros
27Medical School, University of Cyprus, Nicosia, Cyprus
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Siobhán B. Carr
28Primary Ciliary Dyskinesia Centre, Dept of Paediatric Respiratory Medicine, Imperial College and Royal Brompton Hospital, London, UK
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Tables

  • Supplementary Materials
  • TABLE 1

    Clinical trials in primary ciliary dyskinesia (PCD) patients that used a definition for pulmonary exacerbations

    First author [ref.]#Study aimsStudy populationMethod used to develop the definitionDefinition of exacerbation
    Kobbernagel [16]Protocol for randomised controlled trial to determine the efficacy and safety of azithromycin maintenance therapyPCD children aged >7 years and adultsFace-to-face discussion at BESTCILIA study meetingEither
    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 exacerbationsCystic fibrosis and PCD children aged >6 yearsResearcher definedIncrease in respiratory symptoms (not listed) treated with oral antibiotics
    Sunther [15]Recovery of baseline lung function after pulmonary exacerbationPCD childrenResearcher definedA 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 inclusionSurvey 2#Survey 3¶
      calculated ranking
      (1=most important)
      Survey 4+
      % agreement
      Mean score% agree
      Change in sputum volume and/or colour1.5493296
      Increased cough1.64891100
      New/increased haemoptysis1.8679892
      Increased shortness of breath (parent/patient perceived)1.54100392
      Increased respiratory rate2.1464
      Increased chest discomfort/chest pain2.0775
      Malaise, tiredness, fatigue or lethargy2.1468792
      Decreased activity2.54NA
      Decreased exercise tolerance2.1471
      Temperature >38°C1.9682984
      Anorexia or weight loss2.50NA
      Change in physical examination of the chest2.29NA
      Increased crepitations/crackles2.2168
      Increased wheeze2.43NA
      New radiographic changes indicative of a pulmonary infection2.18755
      Decrease in pulmonary function of ≥10% from a previously recorded value (FEV1 % pred or FVC % pred)2.11754
      Raised C-reactive protein2.79NA
      Prolonged erythrocyte sedimentation rate3.39NA
      Raised white cell count3.00NA
      Raised neutrophil count2.96NA
      Physician decision to change treatment because of perceived change in condition  692

      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

      • Tables
      • Supplementary Material

        Please note: supplementary material is not edited by the Editorial Office, and is uploaded as it has been supplied by the author.

        Pulmonary Exacerbations Consensus Survey 00147-2018_supp

        Supplementary tables 00147-2018_supp_tables

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      Pulmonary exacerbations in patients with primary ciliary dyskinesia: an expert consensus definition for use in clinical trials
      Jane S. Lucas, Florian Gahleitner, Adelina Amorim, Mieke Boon, Philippa Brown, Carolina Constant, Simon Cook, Suzanne Crowley, Damien M.S. Destouches, Ernst Eber, Huda Mussaffi, Eric Haarman, Amanda Harris, Cordula Koerner-Rettberg, Claudia E. Kuehni, Philipp Latzin, Michael R. Loebinger, Natalie Lorent, Bernard Maitre, Antonio Moreno-Galdó, Kim G. Nielsen, Uğur Özçelik, Lue Katrine Drasbæk Philipsen, Petr Pohunek, Eva Polverino, Jessica Rademacher, Phil Robinson, Deborah Snijders, Panayiotis Yiallouros, Siobhán B. Carr
      ERJ Open Research Feb 2019, 5 (1) 00147-2018; DOI: 10.1183/23120541.00147-2018

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      Pulmonary exacerbations in patients with primary ciliary dyskinesia: an expert consensus definition for use in clinical trials
      Jane S. Lucas, Florian Gahleitner, Adelina Amorim, Mieke Boon, Philippa Brown, Carolina Constant, Simon Cook, Suzanne Crowley, Damien M.S. Destouches, Ernst Eber, Huda Mussaffi, Eric Haarman, Amanda Harris, Cordula Koerner-Rettberg, Claudia E. Kuehni, Philipp Latzin, Michael R. Loebinger, Natalie Lorent, Bernard Maitre, Antonio Moreno-Galdó, Kim G. Nielsen, Uğur Özçelik, Lue Katrine Drasbæk Philipsen, Petr Pohunek, Eva Polverino, Jessica Rademacher, Phil Robinson, Deborah Snijders, Panayiotis Yiallouros, Siobhán B. Carr
      ERJ Open Research Feb 2019, 5 (1) 00147-2018; DOI: 10.1183/23120541.00147-2018
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