Department of Paediatrics, University Hospital of Pisa, Pisa, Italy.
Early Hum Dev. 2013 Oct;89 Suppl 3:S45-8. doi: 10.1016/j.earlhumdev.2013.07.022. Epub 2013 Aug 22.
Primary ciliary dyskinesia (PCD) is a congenital, clinically and ultrastructurally heterogeneous disease due to abnormal structure and/or function of cilia, with impaired mucociliary transport leading to several respiratory disorders. PCD can be diagnosed by the combination of thorough clinical examination with functional and ultrastructural analysis of the cilia. This paper shows progresses in PCD diagnosis obtained by ciliogenesis in culture evaluation of ciliated respiratory cells and by genetic analysis of mutations in candidate genes. Moreover, since to date no specific treatments are available to correct the ciliary dysfunction, the paper shows the proper therapeutical approach by the use of respiratory physiotherapy and regular exercise to favour airways clearance, by antibiotics administration to control acute airway infections. Macrolides administration as antiinflammatory option is suggested.
原发性纤毛运动障碍(PCD)是一种先天性的、临床和超微结构上具有异质性的疾病,由于纤毛的结构和/或功能异常,导致黏液纤毛传输功能受损,从而引起多种呼吸道疾病。PCD 可以通过彻底的临床检查与纤毛的功能和超微结构分析相结合进行诊断。本文展示了通过培养评估呼吸细胞纤毛的纤毛发生和候选基因突变的遗传分析,在 PCD 诊断方面取得的进展。此外,由于迄今为止尚无可用的特定治疗方法来纠正纤毛功能障碍,本文还展示了通过使用呼吸物理疗法和定期运动来促进气道清除、通过抗生素治疗来控制急性气道感染的适当治疗方法。建议使用大环内酯类药物作为抗炎选择。