Registry Study on Primary Ciliary Dyskinesia in Chinese children-a Multicenter, Prospective Cohort Study
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 100
- 主要终点
- Change from baseline in lung function on the spirometry
研究概览
简要总结
This study is a multicenter, prospective cohort study of patients diagnosed with primary ciliary dyskinesia, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 10 years.
详细描述
All new cases of primary ciliary dyskinesia which was confirmed or diagnosed at each center from the beginning of the study are made the investigation of the clinical manifestations and specific tests by the standard diagnostic process.Then all the patients' following clinical data will be followed for 10 years (once per six month): clinical manifestations, lung function, chest imaging (once per year), quality of life and other indicators.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A included patient must be coincident with all the following items:
- •Age 0~18 years old
- •Any organ system symptoms consistent with PCD and being conform to the clinical diagnostic standard of Katergener syndrome or being coincident with at least two following specific tests:
- •Abnormal ciliary beat frequency or movement by the high speed photography microscope
- •Abnormal ciliary structure through the electronic microscopy
- •The nasal NO decreased significantly
- •The target gene mutation found
- •The clinical diagnostic criteria of the Katergener syndrome: ① bronchial expansion; ② sinusitis or nasal polyps; ③ transposition of viscera and (or) dextrocardia.
- •If all the typical clinical manifestations but only 1 specific test with positive results, can also be included in the registration of suspected PCD cases
- •Consent to provide the related clinical specimen to the certain hospital
- •The guardians of the patients fully understand the purpose of the study, volunteer their children to participate in this study, and sign informed consent.
排除标准
- •Subject will be excluded if she or he has one of the following:
- •It is unable to provide complete medical records or the current condition can not accept the diagnosis process
- •She or he cannot agree to participate in the study.
结局指标
主要结局
Change from baseline in lung function on the spirometry
时间窗: 10 years
forced expiratory volume at one second (FEV1) in Liter
次要结局
未报告次要终点
研究者
Baoping XU
Chief of Respiratory Department
Beijing Children's Hospital