A National Registry on Clinical Manifestations, Genetics, Interventions, and Outcomes in Chinese Patients With Lymphangioleiomyomatosis (LAM-CHINA)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 1,500
- 试验地点
- 1
- 主要终点
- Change in spirometry (FEV1 and FVC) of lymphangioleiomyomatosis patients.
研究概览
简要总结
Pulmonary lymphangioleiomyomatosis (LAM), a disease characterized by diffuse cystic changes in the lung, is a rare disorder that affects almost exclusively women. The main objectives of this study are to accurately evaluate the prevalence of LAM, the status of disease, the diagnosis and treatment, the quality of care, and the health related outcomes in China.
详细描述
Pulmonary lymphangioleiomyomatosis (LAM), a disease characterized by diffuse cystic changes in the lung, is a rare disorder that affects almost exclusively women. The main objectives of this study are to accurately evaluate the prevalence of LAM, the status of disease, the diagnosis and treatment, the quality of care, and the health related outcomes in China. This is a register study lasting 4 years, aims to raise 800 subjects. Primary endpoint is the annual change of forced expiratory volume in the first second (FEV1) and forced vital capacity (FVC) in pulmonary function tests.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Gender: female.
- •Age: no limitation.
- •Diagnosis meets one of the following criteria, (1) definite or probable diagnosis of LAM based on ATS/JRS and ERS criteria. (2) Investigators recommend including of the patient.
排除标准
- •Suspected LAM patients without other supporting evidence for LAM diagnosis.
- •No diffuse cystic lesions in the lung.
- •Patients with bilateral cystic lung lesions but the LAM diagnosis cannot be established.
- •Without signed informed consent.
- •Difficult to follow up.
结局指标
主要结局
Change in spirometry (FEV1 and FVC) of lymphangioleiomyomatosis patients.
时间窗: 10 years
Spirometry will be evaluated at baseline and through study completion, an average of 3 years.
次要结局
- Annual incidence of major health outcomes: hemoptysis, pneumothorax, chylothorax, and spontaneous hemorrhage of kidney angiomyolipoma (AML).(10 years)
- Change of diffusing capacity of the lung.(10 years)
- Other health outcomes: pregnancy, malignant tumors except LAM, lung transplantation, and death.(10 year)
- The incidence of adverse drug reactions of long-time treatment with rapamycin.(10 years)