Czech AATD Registry
Czech Alpha-1 Antitrypsin Deficiency Registry, the National Observational Study.
Alpha-1-antitrypsin deficiency is the most common congenital disease of the respiratory system, leading to early pulmonary emphysema or bronchiectasis. Pulmonary involvement significantly accelerates active cigarette smoking. Patients with alpha-1-antitrypsin deficiency may also have liver cirrhosis, vasculitis, skin or intestinal disorders.
The AATD Registry is a non-interventional multicenter retrospective prospective longitudinal follow-up of patients with alpha-1-antitrypsin deficiency.
The aim of the AATD National Registry is to collect and analyze clinical data in patients with alpha-1 antitrypsin deficiency.
研究概览
地位
条件
详细说明
Alpha-1 antitrypsin deficiency is a genetic disorder that may result in lung disease or liver disease. It is assume that it affects 1 person from a cohort of 2,000-5,000 people of the general population. Among patients with COPD, the incidence of the disorder is significantly higher. The prognosis of these patients is incomparably worse compared to classic COPD, because it affects younger patients and the rate of lung tissue loss is faster. The diagnosis is made in patients with pre-existing COPD by examination of the plasma concentration of AAT. In case of its reduction, genetic examination is added. The progression of the disease is rapid and has been shown to be slowed by lifelong augmentation treatment with human AAT. However, in routine clinical practice, it is very difficult to assess the effectiveness of treatment, the progression of lung disease or the prognosis of the disease.
The AATD registry is a non-interventional multicenter retrospective prospective longitudinal follow-up of patients with alpha-1-antitrypsin deficiency. The national registry collects data from all patients with severe or rare AAT deficiency, regardless of the type of organ impairment and age, and thus provides a view of this genetic variation in the Czech population.
The aim of the AATD Registry is to collect and analyse clinical data of patients with alpha-1 antitrypsin deficiency and increase the professional awareness of this hereditary disease.
研究类型
注册 (预期的)
联系人和位置
学习联系方式
- 姓名:Katerina Kusalova, Ing
- 电话号码:+420723949465
- 邮箱:kusalova@biostatistika.cz
学习地点
-
-
Czech Republic
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Praha、Czech Republic、捷克语、14059
- 招聘中
- Thomayer University Hospital
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首席研究员:
- Jan Chlumsky, MD
-
-
参与标准
资格标准
适合学习的年龄
- 孩子
- 成人
- 年长者
接受健康志愿者
有资格学习的性别
取样方法
研究人群
描述
Inclusion Criteria:
- Patients with alpha-1-antitrypsin deficiency
Exclusion Criteria:
- Patient disagreement with inclusion in the study
学习计划
研究是如何设计的?
设计细节
研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
---|---|---|
Changes of lung function parameters over time
大体时间:within one year after completion
|
assessement of the rate of decline of FEV1 (ml, %predicted), measured annually
|
within one year after completion
|
Changes of exercise tolerance tolerance over time
大体时间:within one year after completion
|
assessment of changes of peak oxygen consumption (peakVO2, ml/kg/min) measured every two years
|
within one year after completion
|
Changes of respiratory function over time
大体时间:within one year after completion
|
assessement of the rate of decline of TLco (mol/min/kPa, %predicted), measured annually
|
within one year after completion
|
Changes of quality of life over time
大体时间:within one year after completion
|
assessement of the rate of decline of COPD assessment test (CAT, points), measured annually
|
within one year after completion
|
Relationship of pulmonary function and lung CT densitometry to better determine phenotypes of COPD due to AAT deficiency
大体时间:within one year after completion
|
Assessement of any possible relationship of primary outcomes 1-3 using LAA (low attenuation area, %) and distribution of emphysema (craniocaudal distribution of emhysema, points)
|
within one year after completion
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
---|---|---|
Behavior of individuals with no or minimal lung involvement
大体时间:within one year after completion
|
Assessement of prognosis of deficient non-smokers by monitoring primary outcome parameters 1-3
|
within one year after completion
|
Progression of other organ disorders, namely liver
大体时间:within one year after completion
|
Laboratory detection of changes in liver tests (ALT, AST, ALP, GGT, ukat/l)
|
within one year after completion
|
合作者和调查者
调查人员
- 首席研究员:Jan MD Chlumsky, PhD、Thomayer University Hospital
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (预期的)
研究完成 (预期的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他研究编号
- IBA1115
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