International Primary Ciliary Dyskinesia Cohort
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 3,400
- 试验地点
- 1
- 主要终点
- BMI
研究概览
简要总结
The iPCD Cohort is an international cohort that assembles available retrospective datasets and prospectively newly collected clinical and diagnostic data from patients suffering from primary ciliary dyskinesia (PCD) worldwide, to answer pertinent questions on clinical phenotype, disease severity, prognosis and effect of treatments in patients with this rare multiorgan disease.
详细描述
The iPCD Cohort was set up under the framework of the European Union (EU) funded 7th Framework Programme (FP7) project Better Experimental Screening and Treatment for Primary Ciliary Dyskinesia (BESTCILIA). The iPCD Cohort is hosted at the Institute of Social and Preventive Medicine at the University of Bern, Switzerland. Research is performed in close collaboration with all data contributors.
Aims:
This combined international dataset allows investigation of PCD epidemiology in a large international study population in order to: 1) describe the spectrum of clinical phenotypes and disease severity in PCD patients by age, sex and time period of diagnosis; 2) describe short-term and long-term prognosis of PCD, looking at important outcomes such as growth, lung function and respiratory failure, bacterial colonisation, hearing loss, fertility, and mortality; and 3) identify predictors of long-term outcomes such as age at diagnosis, clinical phenotype, ultrastructural defects, genotype and clinical care.
Study design:
The iPCD Cohort is an international cohort, combining available data on PCD from national or local registries and clinical or diagnostic databases. All participating centres delivered retrospectively collected data; new centres joining the iPCD Cohort in the future can also participate with retrospectively and prospectively collected data.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed with primary ciliary dyskinesia
排除标准
- 未提供
结局指标
主要结局
BMI
时间窗: every 3 months up to 10 years
Body Mass Index (BMI) z-scores calculated based on available national and international references
Microbiology results
时间窗: every 3 months up to 10 years
Results of microbiology cultures of respiratory samples (sputum, cough swabs, throat swabs, ear swabs, bronchoalveolar lavage) and information on antibiotic resistance (in positive cultures)
Height
时间窗: every 3 months up to 10 years
Height z-scores calculated based on available national and international references
Lung function measurements
时间窗: every 3 months up to 10 years
Spirometric indices, particularly Forced expiratory volume in 1 sec (FEV1) and Forced vital capacity (FVC) z-scores calculated based on Global Lung Function Initiative (GLI) reference values
Diagnostic test results
时间窗: at diagnosis/ study entry
Results of performed PCD diagnostic tests including measurement of nasal nitric oxide, electron microscopy findings, beat frequency and pattern.
Imaging results
时间窗: every 3 months up to 10 years
Radiological findings from sinus and lung imaging tests including x-rays, computed tomography and magnetic resonance imaging
Clinical symptoms and signs
时间窗: every 3 months up to 10 years
Prevalence of reported clinical symptoms at different age groups, including rhinitis, cough, otitis, sinusitis, pneumonia, laterality defects, congenital heart disease and fertility problems.
次要结局
未报告次要终点