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ERJ Open Res. 2020 Feb 10;6(1). pii: 00237-2019. doi: 10.1183/23120541.00237-2019. eCollection 2020 Jan.

Standardised clinical data from patients with primary ciliary dyskinesia: FOLLOW-PCD.

Author information

1
Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
2
Paediatric Respiratory Medicine, Children's University Hospital of Bern, University of Bern, Bern, Switzerland.
3
AP-HP, Hôpital Kremlin-Bicetre, Service d'ORL et de Chirurgie Cervico-Faciale, Le Kremlin-Bicêtre, France.
4
Faculté de Médecine, Université Paris-Saclay, Le Kremlin-Bicêtre, France.
5
INSERM, U955, Créteil, France.
6
CNRS, ERL 7240, Créteil, France.
7
Dept of Paediatrics, University Hospital Gasthuisberg, Leuven, Belgium.
8
Division of Paediatric Pulmonology and Allergology, Dept of Paediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria.
9
Service de Génétique et Embryologie Médicales, Hôpital Armand-Trousseau, Assistance Publique-Hôpitaux de Paris, Paris, France.
10
Primary Ciliary Dyskinesia Centre, NIHR Respiratory Biomedical Research Centre, University of Southampton, Southampton, UK.
11
Primary Ciliary Dyskinesia Centre, Royal Brompton and Harefield Foundation Trust, London, UK.
12
Assistance Publique-Hôpitaux de Paris, Hôpital Cochin, Service de Pneumologie, Paris, France.
13
Division of Respiratory Medicine, University Children's Hospital of Zurich, Zurich, Switzerland.
14
Dept of Pediatric Pulmonology, Marmara University, School of Medicine, Istanbul, Turkey.
15
Dept of Paediatric Pneumology, University Children's Hospital of Ruhr University Bochum, Bochum, Germany.
16
Centre Hospitalier Intercommunal de Créteil, Service de Pneumologie et de Pathologie Professionnelle, DHU A-TVB, Université Paris Est-Créteil, Créteil, France.
17
INSERM U955, Institut Mondor de Recherche Biomédicale (IMRB) Equipe 04, Créteil, France.
18
Danish PCD Centre Copenhagen, Paediatric Pulmonary Service, Copenhagen University Hospital, Copenhagen, Denmark.
19
Al-Quds University, Faculty of Medicine, East Jerusalem, Palestine.
20
Leeds Teaching Hospital NHS Foundation Trust Leeds, Leeds, UK.
21
Tayside Respiratory Research Group, University of Dundee, Dundee, UK.
22
Paediatric Pulmonary Dept, Trousseau Hospital APHP, Sorbonne Universities and Pierre et Marie Curie University, Paris, France.
23
Sorbonne Université, INSERM, Centre de Recherche Saint-Antoine, CRSA, Paris, France.

Abstract

Clinical data on primary ciliary dyskinesia (PCD) are limited, heterogeneous and mostly derived from retrospective chart reviews, leading to missing data and unreliable symptoms and results of physical examinations. We need standardised prospective data collection to study phenotypes, severity and prognosis and improve standards of care. A large, international and multidisciplinary group of PCD experts developed FOLLOW-PCD, a standardised clinical PCD form and patient questionnaire. We identified existing forms for clinical data collection via the Better Experimental Approaches to Treat PCD (BEAT-PCD) COST Action network and a literature review. We selected and revised the content items with the working group and patient representatives. We then revised several drafts in an adapted Delphi process, refining the content and structure. FOLLOW-PCD has a modular structure, to allow flexible use based on local practice and research focus. It includes patient-completed versions for the modules on symptoms and lifestyle. The form allows a comprehensive standardised clinical assessment at baseline and for annual reviews and a short documentation for routine follow-up. It can either be completed using printable paper forms or using an online REDCap database. Data collected in FOLLOW-PCD version 1.0 is available in real-time for national and international monitoring and research. The form will be adapted in the future after extensive piloting in different settings and we encourage the translation of the patient questionnaires to multiple languages. FOLLOW-PCD will facilitate quality research based on prospective standardised data from routine care, which can be pooled between centres, to provide first-line and real-time evidence for clinical decision-making.

Conflict of interest statement

Conflict of interest: M. Goutaki has nothing to disclose. Conflict of interest: J-F. Papon has nothing to disclose. Conflict of interest: M. Boon reports COST BEAT-PCD Action (BM 1407) during the conduct of the study, and Postdoc KOOR funding from the University Hospital Leuven and MyCyFAPP Horizon 2020 EU funding outside the submitted work. Conflict of interest: C. Casaulta has nothing to disclose. Conflict of interest: E. Eber has nothing to disclose. Conflict of interest: E. Escudier has nothing to disclose. Conflict of interest: F.S. Halbeisen has nothing to disclose. Conflict of interest: A. Harris has nothing to disclose. Conflict of interest: C. Hogg has nothing to disclose. Conflict of interest: I. Honore has nothing to disclose. Conflict of interest: A. Jung has nothing to disclose. Conflict of interest: B. Karadag has nothing to disclose. Conflict of interest: C. Koerner-Rettberg has nothing to disclose. Conflict of interest: M. Legendre has nothing to disclose. Conflict of interest: B. Maitre has nothing to disclose. Conflict of interest: K.G. Nielsen has nothing to disclose. Conflict of interest: B. Rubbo has nothing to disclose. Conflict of interest: N. Rumman has nothing to disclose. Conflict of interest: L. Schofield has nothing to disclose. Conflict of interest: A. Shoemark has nothing to disclose. Conflict of interest: G. Thouvenin has nothing to disclose. Conflict of interest: H. Wilkins has nothing to disclose. Conflict of interest: J.S. Lucas reports COST Action BM1407 BEAT-PCD during the conduct of the study. Conflict of interest: C.E. Kuehni has nothing to disclose.

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