Primary ciliary dyskinesia presentation in 60 children according to ciliary ultrastructure

Eur J Pediatr. 2013 Aug;172(8):1053-60. doi: 10.1007/s00431-013-1996-5. Epub 2013 Apr 10.

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • 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

Substances

  • Dyneins