Clinical trial NCT03193892: A National Registry on Chinese Patients With Lymphangioleiomyomatosis
Official title: A National Registry on Clinical Manifestations, Genetics, Interventions, and Outcomes in Chinese Patients With Lymphangioleiomyomatosis (LAM-CHINA)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Pulmonary Function
Keywords: registry
China
lymphangioleiomyomatosis
Lead sponsor: Peking Union Medical College Hospital
Sponsor class: OTHER
Collaborators: name: Shanghai Zhongshan Hospital
name: Guangzhou Institute of Respiratory Disease
name: Central South University
name: Anhui Provincial Hospital
name: Xiangya Hospital of Central South University
name: The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
name: Wuxi People's Hospital
name: Beijing Shijitan Hospital, Capital Medical University
name: West China Hospital
name: Fujian Provincial Hospital
name: Wuhan TongJi Hospital
First posted: 2017-06-21
Enrollment: 1500
Sex: FEMALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Study Population is from across the country of China.

Brief summary: 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: 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.

Eligibility criteria: Inclusion Criteria:

1. Gender: female.
2. Age: no limitation.
3. 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:

1. Suspected LAM patients without other supporting evidence for LAM diagnosis.
2. No diffuse cystic lesions in the lung.
3. Patients with bilateral cystic lung lesions but the LAM diagnosis cannot be established.
4. Without signed informed consent.
5. Difficult to follow up.

Primary outcomes:
measure: Change in spirometry (FEV1 and FVC) of lymphangioleiomyomatosis patients.
timeFrame: 10 years
Secondary outcomes:
measure: Change of diffusing capacity of the lung.
timeFrame: 10 years
measure: Annual incidence of major health outcomes: hemoptysis, pneumothorax, chylothorax, and spontaneous hemorrhage of kidney angiomyolipoma (AML).
timeFrame: 10 years
measure: Other health outcomes: pregnancy, malignant tumors except LAM, lung transplantation, and death.
timeFrame: 10 year
measure: The incidence of adverse drug reactions of long-time treatment with rapamycin.
timeFrame: 10 years
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
Study officials:
References: