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Primary ciliary dyskinesia (PCD) is a rare genetic disorder characterised by impaired mucociliary clearance leading to irreversible lung damage. In contrast to other rare lung diseases like cystic fibrosis (CF), there are only few clinical trials and limited evidence-based treatments. Management is mainly based on expert opinions and treatment is challenging due to a wide range of clinical manifestations and disease severity. To improve clinical and translational research and facilitate development of new treatments, the clinical trial network for PCD (PCD-CTN) was founded in 2020 under the framework of the European Reference Network (ERN)-LUNG PCD Core. Applications from European PCD sites interested in participating in the PCD-CTN were requested. Inclusion criteria consisted of patient numbers, membership of ERN-LUNG PCD Core, use of associated standards of care, experience in PCD and/or CF clinical research, resources to run clinical trials, good clinical practice (GCP) certifications and institutional support. So far, applications from 22 trial sites in 18 European countries have been approved, including >1400 adult and >1600 paediatric individuals with PCD. The PCD-CTN is headed by a coordinating centre and consists of a steering and executive committee, a data safety monitoring board and committees for protocol review, training and standardisation. A strong association with patient organisations and industrial companies are further cornerstones. All participating trial sites agreed on a code of conduct. As CTNs from other diseases have demonstrated successfully, this newly formed PCD-CTN operates to establish evidence-based treatments for this orphan disease and to bring new personalised treatment approaches to patients., Competing Interests: Conflict of interest: J. Raidt declares grants for Clinical Research Unit “Male Germ Cells: from Genes to Function” (CRU 326) – Subproject RA3522/1-1 from the Deutsche Forschungsgemeinschaft, in connection with the present manuscript; that they are a holder of patent WO 2020/165352 A1 (Treatment of ciliopathies); and an unpaid role as a Deputy Director of PCD-CTN. Conflict of interest: B. Maitre declares unpaid roles as a Deputy Director of PCD-CTN and as a member of the scientific committee of RADICO PCD. Conflict of interest: P. Pennekamp declares research grants to their institution from NEOCYST (BMBF, 01GM1903A); and that they are a holder of patent WO 2020/165352 A1 (Treatment of ciliopathies). Conflict of interest: M. Armengot declares research grant FIS PI19/00949 from Instituto de Salud Carlos III, in connection with the present manuscript. Conflict of interest: M. Boon declares Forton grant 2020-J1810150-217926 from the King Baudouin Foundation, in the 36 months prior to manuscript submission. Conflict of interest: S.B. Carr declares grants to their institution from the National Institute of Health Research (Programme Development Grant 202952; Health Technology Assessment Grant NIHR121889; HTA grant 14/22/23; NIHR RfPb QOL-PCD; NIHR RfPb VALU- CF); consulting fees paid to their institution by Vertex Pharmaceuticals; personal and institutional honoraria from Chiesi Pharmaceuticals; payment to their institution for participation on a Data Safety Monitoring Board or Advisory Board from Profile Pharma, Pharmaxis and Vertex Pharmaceuticals, all in the 36 months prior to manuscript submission; and that they are Chair of the UK CF Registry Steering Committee (payment to their institution) and of the European CF Society Patient Registry Scientific Committee (unpaid). Conflict of interest: M.A. Mall declares research grant 82DZL009B1 from the German Federal Ministry of Education and Research, in connection with the present manuscript. Conflict of interest: M. Narayanan declares that they have been a co-applicant on the National Institute for Health Research – Research for patient benefit (NIHR –RfPB) grant for ‘Parent reported quality of life measures for young children with primary ciliary dyskinesia (QOL-PCD study)’ in the 36 months prior to manuscript submission; and that they are a member of the British Paediatric Respiratory Society executive committee and the British Thoracic Society standards of care committee. Conflict of interest: P. Pohunek declares research grants from the Ministry of Health of the Czech Republic (NV19-07-00210) and Charles University Grant Agency (670119P) in connection with the present manuscript. Conflict of interest: S. Range declares an unpaid role as medical advisor to PCD Support. Conflict of interest: F.C. Ringshausen declares grants to their institution from the German Center for Lung Research (DZL), the German Center for Infection Research (DZIF), IMI (EU/EFPIA), iABC Consortium (including Alaxia, Basilea, Novartis and Polyphor), Mukoviszidose Institute, Novartis, Insmed Germany, Grifols, Bayer and InfectoPharm; consulting fees from Parion, Grifols, Zambon, Insmed and the Helmholtz-Zentrum für Infektionsforschung; payments or honoraria from I!DE Werbeagentur GmbH; Interkongress GmbH; AstraZeneca; Insmed; Grifols and Universitätsklinikum Frankfurt am Main; payment to their institution for expert testimony at the Social Court Cologne; support for attending meetings from German Kartagener Syndrome and PCD PAG and Mukoviszidose e.V; participation on a Data Safety Monitoring Board or Advisory Board for Insmed, Grifols and Shionogi; fees for clinical trial participation paid to their institution by Abbvie, AstraZeneca, Boehringer Ingelheim, Celtaxsys, Corbus, Insmed, Novartis, Parion, University of Dundee, Vertex and Zambon; and honorary roles as Coordinator of the ERN-LUNG Bronchiectasis Core Network, Chair of the German Bronchiectasis Registry PROGNOSIS, Member of the SteerCo of the European Bronchiectasis Registry EMBARC, Member of the SteerCo of the European NTM Registry EMBARC-NTM, Co-Speaker of the Medical Advisory Board of the German Kartagener Syndrome and PCD PAG, Speaker of the Respiratory Infections and TB group of the German Respiratory Society (DGP), Speaker of the Cystic Fibrosis group of German Respiratory Society (DGP), PI of the DZL, member of the protocol review committee of the PCD-CTN and Member of Physician Association of the German Cystic Fibrosis PAG. Conflict of interest: J. Roehmel declares payments or honoraria from Vertex Pharmaceuticals, in the 36 months prior to manuscript submission. Conflict of interest: G. Thouvenin declares unpaid membership of the RADICO PCD scientific committee. Conflict of interest: H. Omran declares grants for Clinical Research Unit “Male Germ Cells: from Genes to Function” (CRU 326) – Subproject RA3522/1-1 from the Deutsche Forschungsgemeinschaft, from Interdisziplinaeres Zentrum für Klinische Forschung Muenster (Om2/009/12; Om2/015/16; Om2/010/20), from BESTCILIA (EU FP7 GA 305404) and from Registry Warehouse (Horizon2020 GA 777295), all in connection with the present manuscript; in addition to grants to their institution from LYSOCIL (Horizon2020 GA n°811087) NEOCYST (BMBF, 01GM1903A) and Transkripttherapie für primäre ziliäre Dyskinesien (Zentrales Innovationsprojekt Mittelstand (ZIM), BMWi; ZF-4610102SK8), in the 36 months prior to manuscript submission; that they are a holder of patent WO 2020/165352 A1 (Treatment of ciliopathies); and unpaid roles as Head of the ERN-LUNG PCD Core and as a member of the PCD Family Support Group medical advisory board. Conflict of interest: K.G. Nielsen declares funding for the present manuscript from the Danish Children's Lung Foundation (Børnelungefonden); and an unpaid role as a Director of the PCD-CTN. Conflict of interest: All other authors declare no competing interests., (Copyright ©The authors 2022.)

Introduction: In primary ciliary dyskinesia (PCD) impaired mucociliary clearance leads to recurrent airway infections and progressive lung destruction, and concern over chronic airway infection and patient-to-patient transmission is considerable. So far, there has been no defined consensus on how to control infection across centres caring for patients with PCD. Within the BEAT-PCD network, COST Action and ERS CRC together with the ERN-Lung PCD core a first initiative has now been taken towards creating such a consensus statement., Methods: A multidisciplinary international PCD expert panel was set up to create a consensus statement for infection prevention and control (IP&C) for PCD, covering diagnostic microbiology, infection prevention for specific pathogens considered indicated for treatment and segregation aspects. Using a modified Delphi process, consensus to a statement demanded at least 80% agreement within the PCD expert panel group. Patient organisation representatives were involved throughout the process., Results: We present a consensus statement on 20 IP&C statements for PCD including suggested actions for microbiological identification, indications for treatment of Pseudomonas aeruginosa , Burkholderia cepacia and nontuberculous mycobacteria and suggested segregation aspects aimed to minimise patient-to-patient transmission of infections whether in-hospital, in PCD clinics or wards, or out of hospital at meetings between people with PCD. The statement also includes segregation aspects adapted to the current coronavirus disease 2019 (COVID-19) pandemic., Conclusion: The first ever international consensus statement on IP&C intended specifically for PCD is presented and is targeted at clinicians managing paediatric and adult patients with PCD, microbiologists, patient organisations and not least the patients and their families., Competing Interests: Conflict of interest: J.K Marthin has nothing to disclose. Conflict of interest: J.S. Lucas has nothing to disclose. Conflict of interest: M. Boon has nothing to disclose. Conflict of interest: C. Casaulta has nothing to disclose. Conflict of interest: S. Crowley has nothing to disclose. Conflict of interest: D.M.S. Destouches has nothing to disclose. Conflict of interest: E. Eber has nothing to disclose. Conflict of interest: A. Escribano has nothing to disclose. Conflict of interest: E. Haarman has nothing to disclose. Conflict of interest: C. Hogg has nothing to disclose. Conflict of interest: B. Maitre has nothing to disclose. Conflict of interest: G. Marsh has nothing to disclose. Conflict of interest: V. Martinu has nothing to disclose. Conflict of interest: A. Moreno-Galdó reports a personal payment for a lecture from Abbvie, travel support from Abbvie and Novartis, and personal payments for advisory boards from Abbvie and Sanofi. Conflict of interest: H. Mussaffi has nothing to disclose. Conflict of interest: H. Omran has nothing to disclose. Conflict of interest: P. Pohunek has nothing to disclose. Conflict of interest: B. Rindlisbacher has nothing to disclose. Conflict of interest: P. Robinson has nothing to disclose. Conflict of interest: D. Snijders has nothing to disclose. Conflict of interest: W.T. Walker has nothing to disclose. Conflict of interest: P. Yiallouros has nothing to disclose. Conflict of interest: H.K. Johansen has nothing to disclose. Conflict of interest: K.G. Nielsen has nothing to disclose., (Copyright ©The authors 2021.)

Pulmonary exacerbations are a cause of significant morbidity in patients with primary ciliary dyskinesia (PCD) and are frequently used as an outcome measure in clinical research into chronic lung diseases. So far, there has been no consensus on the definition of pulmonary exacerbations in PCD. 30 multidisciplinary experts and patients developed a consensus definition for children and adults with PCD. Following a systematic review, the panel used a modified Delphi process with a combination of face-to-face meetings and e-surveys to develop a definition that can be used in research settings for children and adults with PCD. A pulmonary exacerbation was defined by the presence of three or more of the following seven items: 1) increased cough, 2) change in sputum volume and/or colour, 3) increased shortness of breath perceived by the patient or parent, 4) decision to start or change antibiotic treatment because of perceived pulmonary symptoms, 5) malaise, tiredness, fatigue or lethargy, 6) new or increased haemoptysis, and 7) temperature >38°C. The consensus panel proposed that the definition should be used for future clinical trials. The definition should be validated and the usability assessed during these studies., Competing Interests: Conflict of interest: J.S. Lucas reports grants, personal fees and nonfinancial support from Aerocrine/Circassia, grants and personal fees from Vertex, and grants from Parion, outside the submitted work. Conflict of interest: F. Gahleitner has nothing to disclose. Conflict of interest: A. Amorim reports personal fees from Zambon and personal fees from Vertex, outside the submitted work. Conflict of interest: M. Boon reports a postdoctoral research grant from KOOR, University Hospital Leuven, outside the submitted work. Conflict of interest: P. Brown has nothing to disclose. Conflict of interest: C. Constant has nothing to disclose. Conflict of interest: S. Cook has nothing to disclose. Conflict of interest: S. Crowley has nothing to disclose. Conflict of interest: D.M.S. Destouches has nothing to disclose. Conflict of interest: E. Eber has nothing to disclose. Conflict of interest: H. Mussaffi has nothing to disclose. Conflict of interest: E. Haarman has nothing to disclose. Conflict of interest: A. Harris has nothing to disclose. Conflict of interest: C. Koerner-Rettberg has nothing to disclose. Conflict of interest: C.E. Kuehni has nothing to disclose. Conflict of interest: P. Latzin reports personal fees from Vertex, Novartis, Roche, Polyphor, Vifor, Gilead, Schwabe, Zambon, Santhera and Vertex, outside the submitted work. Conflict of interest: M.R. Loebinger reports personal fees from Bayer, Griffols, Polyphor and Raptor, and other support from Parion, outside the submitted work. Conflict of interest: N. Lorent reports personal fees from Chiesi, and nonfinancial support from Insmed and Mylan, outside the submitted work. Conflict of interest: B. Maitre has nothing to disclose. Conflict of interest: A. Moreno-Galdó reports personal fees from AbbVie, and other support from AbbVie, Actelion and Novartis, outside the submitted work. Conflict of interest: K.G. Nielsen has nothing to disclose. Conflict of interest: U. Özçelik has nothing to disclosure Conflict of interest: L.K.D. Philipsen has nothing to disclose. Conflict of interest: P. Pohunek reports personal fees from Novartis, Sandoz and Teva, grants from Teva, and personal fees from Stallergenes, S&D Pharma, ALK and Boehringer Ingelheim, outside the submitted work. Conflict of interest: E. Polverino reports personal fees from Bayer, Insmed and Grifols, Zambon, grants and personal fees from Chiesi, and personal fees from Polyphor, during the conduct of the study; none of these fees and grants were strictly related with the topic of this manuscript. Conflict of interest: J. Rademacher has nothing to disclose. Conflict of interest: P. Robinson has nothing to disclose. Conflict of interest: D. Snijders has nothing to disclose. Conflict of interest: P. Yiallouros has nothing to disclose. Conflict of interest: S.B. Carr reports personal fees and other support from Vertex Pharmaceuticals, other support from Chiesi Ltd, personal fees from Teva Pharmaceutical Industries and other support from Pharmaxis Pharmaceuticals Ltd, outside the submitted work.