An international registry for primary ciliary dyskinesia
Primary ciliary dyskinesia (PCD) is a rare autosomal recessive disorder leading to chronic upper and lower airway disease. Fundamental data on epidemiology, clinical presentation, course and treatment strategies are lacking in PCD. We have established an international PCD registry to realise an unme...
Saved in:
| Main Authors: | , |
|---|---|
| Format: | Article (Journal) |
| Language: | English |
| Published: |
2016
|
| In: |
The European respiratory journal
Year: 2016, Volume: 47, Issue: 3, Pages: 849-859 |
| ISSN: | 1399-3003 |
| DOI: | 10.1183/13993003.00776-2015 |
| Online Access: | Verlag, Volltext: http://dx.doi.org/10.1183/13993003.00776-2015 Verlag, Volltext: http://erj.ersjournals.com/content/47/3/849 
|
| Author Notes: | Claudius Werner, Martin Lablans, Maximilian Ataian, Johanna Raidt, Julia Wallmeier, Jörg Große-Onnebrink, Claudia E. Kuehni, Eric G. Haarman, Margaret W. Leigh, Alexandra L. Quittner, Jane S. Lucas, Claire Hogg, Michal Witt, Kostas N. Priftis, Panayiotis Yiallouros, Kim G. Nielsen, Francesca Santamaria, Frank Ückert and Heymut Omran |
| Summary: | Primary ciliary dyskinesia (PCD) is a rare autosomal recessive disorder leading to chronic upper and lower airway disease. Fundamental data on epidemiology, clinical presentation, course and treatment strategies are lacking in PCD. We have established an international PCD registry to realise an unmet need for an international platform to systematically collect data on incidence, clinical presentation, treatment and disease course. The registry was launched in January 2014. We used internet technology to ensure easy online access using a web browser under www.pcdregistry.eu. Data from 201 patients have been collected so far. The database is comprised of a basic data form including demographic and diagnostic information, and visit forms designed to monitor the disease course. To establish a definite PCD diagnosis, we used strict diagnostic criteria, which required two to three diagnostic methods in addition to classical clinical symptoms. Preliminary analysis of lung function data demonstrated a mean annual decline of percentage predicted forced expiratory volume in 1 s of 0.59% (95% CI 0.98-0.22). Here, we present the development of an international PCD registry as a new promising tool to advance the understanding of this rare disorder, to recruit candidates for research studies and ultimately to improve PCD care. Tweetable abstract ERSpublications click to tweetA registry to systematically collect data on clinical presentation, disease course and treatment of PCD http://ow.ly/TtGAR |
|---|---|
| Item Description: | Published online February 29, 2016 First published online: December 09 2015 Gesehen am 19.06.2018 |
| Physical Description: | Online Resource |
| ISSN: | 1399-3003 |
| DOI: | 10.1183/13993003.00776-2015 |