Imaging Biomarkers in Lymphangioleiomyomatosis Clinical Trials. A Wolf in Sheep's Clothing?

Authors

George R Washko
Grace Parraga

Document Type

Article

Publication Date

3-1-2016

Journal

Annals of the American Thoracic Society

Volume

13

Issue

3

First Page

307

Last Page

308

URL with Digital Object Identifier

https://doi.org/10.1513/AnnalsATS.201512-818ED

Abstract

Lymphangioleiomyomatosis (LAM) is a rare disease of premenopausal women. Pulmonary manifestations include cystic destruction of the parenchyma and chylous pleural effusions (1, 2). Patients suffering from this condition experience progressive expiratory airflow obstruction and hypoxemia, which can lead to respiratory failure and death (3, 4). The need to deepen our understanding of LAM and improve patient care is clear. The great challenge to such efforts has been the size of patient cohorts available for clinical investigation. Recognition of this limitation has fostered interest in biomarkers that may be used as intermediate study endpoints for therapeutic trials.

Notes

This is an author-accepted version of the manuscript initially published in the Annals of the American Thoracic Society. The final published version is available at: https://doi.org/10.1513/AnnalsATS.201512-818ED

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