Primary ciliary dyskinesia presentation in 60 children according to ciliary ultrastructure.

Details

Serval ID
serval:BIB_05A34C02613C
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Primary ciliary dyskinesia presentation in 60 children according to ciliary ultrastructure.
Journal
European journal of pediatrics
Author(s)
Vallet C., Escudier E., Roudot-Thoraval F., Blanchon S., Fauroux B., Beydon N., Boulé M., Vojtek A.M., Amselem S., Clément A., Tamalet A.
ISSN
1432-1076 (Electronic)
ISSN-L
0340-6199
Publication state
Published
Issued date
08/2013
Peer-reviewed
Oui
Volume
172
Number
8
Pages
1053-1060
Language
english
Notes
Publication types: Journal Article ; Research Support, Non-U.S. Gov't
Publication Status: ppublish
Abstract
Primary ciliary dyskinesia (PCD) is an inherited disease related to ciliary dysfunction, with heterogeneity in clinical presentation and in ciliary ultrastructural defect. Our study intended to determine if there are phenotypic differences in patients with PCD based on ciliary ultrastructural abnormality. In this retrospective study carried out among 60 children with a definitive diagnosis of PCD, we analyzed clinical, radiological, and functional features at diagnosis and at last recorded visit, according to cilia defect (absence of dynein arms: DAD group, n = 36; abnormalities of the central complex: CCA group, n = 24). Onset of respiratory symptoms occurred later in the CCA than in the DAD group (9.5 versus 0.5 months, p = 0.03). Situs inversus was only observed in the DAD group, while respiratory disease in siblings were more frequent in the CCA group (p = 0.003). At diagnosis, clinical presentation was more severe in the CCA group: frequency of respiratory tract infections (p = 0.008), rhinosinusitis (p = 0.02), otitis complications (p = 0.0001), bilateral bronchiectasis (p = 0.04), and number of hypoxemic patients (p = 0.03). Pulmonary function remained stable in both groups, but outcome was better in the CCA than in the DAD group: less antibiotic therapy and hypoxemic patients (p = 0.004). In conclusion, our results underlined the relationship between the severity of clinical presentation and the ultrastructural ciliary defect.
Keywords
Adolescent, Bronchiectasis/etiology, Child, Child, Preschool, Cilia/pathology, Cilia/ultrastructure, Dyneins/ultrastructure, Female, Humans, Kartagener Syndrome/complications, Kartagener Syndrome/pathology, Male, Microscopy, Electron, Respiratory Function Tests, Respiratory Tract Infections/etiology, Retrospective Studies, Spirometry, Statistics, Nonparametric, Tomography, X-Ray Computed
Pubmed
Web of science
Create date
15/05/2020 17:27
Last modification date
16/05/2020 6:26
Usage data