An international registry for primary ciliary dyskinesia.
Eur Respir J
; 47(3): 849-59, 2016 Mar.
Article
em En
| MEDLINE
| ID: mdl-26659107
ABSTRACT
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 1â
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.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Sistema de Registros
/
Síndrome de Kartagener
Tipo de estudo:
Incidence_studies
/
Observational_studies
/
Prognostic_studies
Limite:
Adolescent
/
Adult
/
Aged
/
Child
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Child, preschool
/
Female
/
Humans
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Infant
/
Male
/
Middle aged
País/Região como assunto:
America do norte
/
Europa
Idioma:
En
Ano de publicação:
2016
Tipo de documento:
Article