A National Registry on Chinese Patients With Lymphangioleiomyomatosis

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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A National Registry on Clinical Manifestations, Genetics, Interventions, and Outcomes in Chinese Patients With Lymphangioleiomyomatosis (LAM-CHINA)

Conditions
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Pulmonary Function

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Exclusion Criteria: 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.
Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in spirometry (FEV1 and FVC) of lymphangioleiomyomatosis patients. | Spirometry will be evaluated at baseline and through study completion, an average of 3 years. | 10 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change of diffusing capacity of the lung. | Pulmonary function tests will be evaluated at baseline and through study completion, an average of 3 years. | 10 years | 
| Annual incidence of major health outcomes: hemoptysis, pneumothorax, chylothorax, and spontaneous hemorrhage of kidney angiomyolipoma (AML). | The number of occurrences will be recorded through study completion, an average of 3 years. | 10 years | 
| Other health outcomes: pregnancy, malignant tumors except LAM, lung transplantation, and death. | The number of occurrences will be recorded through study completion, an average of 3 years. | 10 year | 
| The incidence of adverse drug reactions of long-time treatment with rapamycin. | Common Terminology Criteria for Adverse Events (CTCAE) will be used to evaluate adverse drug reactions through study completion, an average of 3 years. | 10 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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registry, China, lymphangioleiomyomatosis