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

What this Trial Is About

Pulmonary lymphangioleiomyomatosis (LAM) is a rare lung disease characterized by widespread cystic changes and almost exclusively affects women. This research aims to accurately determine how common LAM is in China and to evaluate the current disease status, diagnostic methods, treatment approaches, quality of care, and health outcomes for patients. The study is observational and will collect data over a period of four years from approximately 800 participants. This study is a national registry that gathers clinical information on Chinese women diagnosed with LAM. It will not involve any experimental treatments but will monitor patients' health and treatments they receive in routine care. The primary outcome is to track the annual change in lung function, specifically measuring forced expiratory volume in one second (FEV1) and forced vital capacity (FVC). Secondary outcomes include changes in lung gas exchange ability, occurrence of serious health events such as bleeding or lung complications, pregnancy outcomes, tumors, lung transplants, deaths, and side effects from long-term rapamycin treatment. Participants will be followed for up to ten years with regular pulmonary function tests and health assessments. The study includes collecting data on major health events and adverse effects related to treatment over this time. Researchers will analyze these data to better understand LAM progression and outcomes in Chinese patients. The study requires participants to provide informed consent and be available for follow-up visits to contribute valuable information about this rare disease.

A National Registry on Chinese Patients With Lymphangioleiomyomatosis