LONGITUDINAL STUDY OF PRIMARY CILIARY DYSKINESIA (PCD STUDY)

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 我们已经建立了一个由4个分散在不同地理位置的临床站点组成的联盟，旨在研究罕见的呼吸道疾病。这项纵向研究方案的目的是确定患有这些疾病之一的原发性睫状肌发育不良(PCD)的参与者的表型特征和肺部疾病关键特征的进展与年龄相关的患病率。在PCD中，纤毛的结构和功能异常导致分泌物清除障碍，进而导致呼吸道、鼻窦和中耳的慢性反复感染。 PCD肺部疾病的临床病程尚不清楚。 这项纵向研究旨在使用肺活量测定法确定5至18岁参与者的PCD肺功能进展率。我们还将系统地跟踪其他结果，包括呼吸道病原体(通过呼吸道培养评估)、呼吸道损伤的发病年龄和进展以及支气管扩张(通过胸部高分辨率断层扫描(HRCT)评估)。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. We have established a consortium of 4 geographically-dispersed clinical sites that are designed to study rare diseases of the airways. The purpose of this longitudinal study protocol is to define age-related prevalence of phenotypic characteristics and the progression of key features of lung diseases in participants with one of these disorders, Primary Ciliary Dyskinsesia (PCD). In PCD, the abnormal structure and function of cilia results in impaired clearance of secretions and consequent obstruction and chronic recurrent infections in the airways, sinuses and middle ears. The clinical course of PCD lung disease is not well defined. This longitudinal study is designed to define a rate of progression of PCD lung function in participants between 5 and 18 year of age using spirometry. We will also systematically track other outcomes, including pathogens of the airways(assessed by respiratory cultures), age at onset and progression of airway damage and bronchiectasis (assessed by high-resolution tomography (HRCT) of the chest.