Objectives

The LAM Registry was established to define the natural history and identify prognostic biomarkers to help guide management and decision-making in patients with LAM.

Background

A LAM Registry was established at six centers by the NHLBI. LAM is a rare multi-system disease that can affect the lungs of young women, is of uncertain cause, is usually progressive, and can cause debilitating lung disease which may be corrected with lung transplantation. Several hundred women with the disease were identified, largely through a LAM Foundation in Cincinnati, Ohio.

Participants

Women with a lymphangioleiomyomatosis diagnosis, ages 18-76.

Disease Classification

pulmonary lymphangioleiomyomatosis and tuberous sclerosis-related lymphangioleiomyomatosis.

Design

The registry had six major clinical centers: Cleveland Clinic Foundation, Mayo Clinic-Rochester, National Heart, Lung, and Blood Institute, National Jewish Medical and Research Center, New England Medical Center, and Stanford University Medical Center. Data and lung tissue collected by the registry were collected to characterize the clinical features and natural history of the disease and to determine the efficacy of lung transplantation in this disorder. Eligible patients were seen yearly for up to 4 years at the Clinical Centers or, in some cases, by individual referring physicians. Tissue collected by the LAM Registry was stored at a central NHLBI repository. Outcome events included measures of pulmonary function (eg, spirometry and, when available, lung volumes and diffusing capacity), arterial blood gases or oximetry, walking and resting oxygen titration, cardiopulmonary stress testing, cause-specific mortality, functional status, and clinical events associated with lung transplantation.

Publications

https://pubmed.ncbi.nlm.nih.gov/16210669/

https://pubmed.ncbi.nlm.nih.gov/18096481/

https://pubmed.ncbi.nlm.nih.gov/23007140/