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

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.
Competing Interests: 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.
(Copyright ©ERS 2020.)