PT  - JOURNAL ARTICLE
AU  - Bruna Rubbo
AU  - Laura Behan
AU  - Corine Driessens
AU  - Rodrigo Athanazio
AU  - Antony Terance Benjamin
AU  - Veerappan Ramamoorthi Ram Ganesh
AU  - Johnny Goodwin
AU  - Bulent Karadag
AU  - Panayiotis Kouis
AU  - Ana Reula Martin
AU  - Kim Nielsen
AU  - Mary Anne Olm
AU  - Heymut Omran
AU  - Jean-Francois Papon
AU  - Andreia Pinto
AU  - Nisreen Rumman
AU  - Deborah Snijders
AU  - Zorica Zivkovic
AU  - Camille Parsons
AU  - Claudia E. Kuehni
AU  - Jane S. Lucas
TI  - Service delivery models for primary ciliary dyskinesia, an international comparison
AID  - 10.1183/13993003.congress-2021.PA3461
DP  - 2021 Sep 05
TA  - European Respiratory Journal
PG  - PA3461
VI  - 58
IP  - suppl 65
4099  - http://erj.ersjournals.com/content/58/suppl_65/PA3461.short
4100  - http://erj.ersjournals.com/content/58/suppl_65/PA3461.full
SO  - Eur Respir J2021 Sep 05; 58
AB  - Background: Service delivery models vary between and within countries, yet the extent and determinants of different approaches to deliver care for patients with primary ciliary dyskinesia (PCD) in the last 12 years have not been assessed. This is particularly important in rare diseases due to geographical barriers and need for specialised equipment and personnel.Methods: We used a mixed-methods approach. We conducted interviews with PCD experts between 2016-2018. Transcripts were coded using thematic analysis. Based on themes identified from the interviews, we developed a survey to quantify similarities and differences between models. This was circulated to healthcare professionals involved in care for PCD patients in 2019. Results were compared to a similar survey conducted in 2009.Results: Fifteen PCD experts were interviewed and 56 experts from 31 countries completed the survey (88% response rate). Availability of diagnostic testing has increased in the last ten years. All centres had either transmission electron microscopy or genetic testing available, the two confirmatory tests according to ERS diagnostic guidelines. However, geographical region and size of PCD centres influenced choice of management strategy, with larger centres prioritising effective and less expensive therapies such as airway clearance, nasal rinsing and hypertonic saline. Prophylactic antibiotics were often prescribed in large centres (69% vs 38% in small centres).Conclusions: The variability of management strategies highlight the need for evidence-based management guidelines for PCD. Our findings can inform the establishment of new centres, improve service in existing centres, and set a wider agenda for rare diseases.FootnotesCite this article as: European Respiratory Journal 2021; 58: Suppl. 65, PA3461.This abstract was presented at the 2021 ERS International Congress, in session “Prediction of exacerbations in patients with COPD”.This is an ERS International Congress abstract. No full-text version is available. Further material to accompany this abstract may be available at www.ers-education.org (ERS member access only).