Imaging Biomarkers in Lymphangioleiomyomatosis Clinical Trials. A Wolf in Sheep's Clothing?
Annals of the American Thoracic Society
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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.
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