A National Registry on Chinese Patients With Lymphangioleiomyomatosis

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

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.

Study Overview

• Status: Recruiting
• Conditions: Pulmonary Function

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.

• Study Type: Observational
• Enrollment (Anticipated): 1500

Contacts and Locations

• Study Contact: Name: Kai-Feng Xu, M.D.; Phone Number: 86-10-69155039; Email: xukf@pumch.cn

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100730
      • Recruiting
      • Peking Union Medical College Hospital
      • Contact: Kai-Feng Xu, M.D.; Phone Number: 86-10-69155039; Email: xukf@pumch.cn
      • Contact: Xinlun Tian, M.D.; Phone Number: 86-10-69155039; Email: xinlun_t@sina.com
      • Principal Investigator: Kai-Feng Xu, M.D.
      • Sub-Investigator: Xinlun Tian, M.D.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Female

• Sampling Method: Probability Sample

Study Population

Study Population is from across the country of China.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital
• Collaborators: Xiangya Hospital of Central South University; Shanghai Zhongshan Hospital; West China Hospital; The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School; Beijing Shijitan Hospital, Capital Medical University; Guangzhou Institute of Respiratory Disease; Central South University; Wuhan TongJi Hospital; Fujian Provincial Hospital; Anhui Provincial Hospital; Wuxi People's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2017
• Primary Completion (Anticipated): October 1, 2026
• Study Completion (Anticipated): October 1, 2026

Study Registration Dates

• First Submitted: June 14, 2017
• First Submitted That Met QC Criteria: June 18, 2017
• First Posted (Actual): June 21, 2017

Study Record Updates

• Last Update Posted (Actual): September 24, 2021
• Last Update Submitted That Met QC Criteria: September 18, 2021
• Last Verified: September 1, 2021

More Information

Terms related to this study

• Keywords: China; registry; lymphangioleiomyomatosis
• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Lymphangiomyoma; Lymphatic Vessel Tumors; Perivascular Epithelioid Cell Neoplasms; Lymphangioleiomyomatosis
• Other Study ID Numbers: 2016YFC091502LAM

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: Currently, there is no such a plan to share IPD to other researchers

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No