A National Registry on Chinese Patients With Lymphangioleiomyomatosis

Conditions

Pulmonary Function

Keywords

registry, China, lymphangioleiomyomatosis

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.

Kuanfeng Xu, Lin Yang, Hexiao Wang, Cheng Cui, Xiao Zhang

American Journal of Respiratory and Critical Care Medicine2025-05-01

10.1164/ajrccm.2025.211.abstracts.a1823

Long-term safety and influence on growth in patients receiving sirolimus: a pooled analysis.

Wang YY, Zou LP, Xu KF, Xu WS, Zhang MN, Lu Q, Tian XL, Pang LY, He W, Wang QH, Gao Y, Liu LY, Chen XQ, Ma SF, Chen HM, Dun S, Yang XY, Luo XM, Huang LL, Li YF.

2024-08-154 citations

10.1186/s13023-024-03243-5

Determinants of Progression and Mortality in Lymphangioleiomyomatosis.

Xu W, Yang C, Cheng C, Wang Y, Hu D, Huang J, He Y, Wang J, Chen K, Yang L, Zhou W, Zhang T, Liu S, Dai J, Meng S, Li X, Yang Y, Wang ST, Feng R, Zhang W, Zhang H, Wang L, Tian X, Xu KF.

2023-02-1822 citations

10.1016/j.chest.2023.02.026

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

FEMALE

Healthy volunteers

No

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.

Design outcomes

Primary

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

Secondary

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

Countries

China

Contacts

Primary ContactKai-Feng Xu, M.D.

xukf@pumch.cn86-10-69155039

Outcome results