Unexpected sirolimus-stimulated airway hyperreactivity in lymphangioleiomyomatosis

Wendy K Steagall; Mario Stylianou; Gustavo Pacheco-Rodriguez; Zu Xi Yu; Joel Moss

doi:10.1183/23120541.00305-2023

Unexpected sirolimus-stimulated airway hyperreactivity in lymphangioleiomyomatosis

ERJ Open Res. 2023 Aug 14;9(4):00305-2023. doi: 10.1183/23120541.00305-2023. eCollection 2023 Jul.

Authors

Wendy K Steagall¹, Mario Stylianou², Gustavo Pacheco-Rodriguez¹, Zu Xi Yu³, Joel Moss¹

Affiliations

¹ Pulmonary Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
² Office of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
³ Pathology Facility, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.

Abstract

Lymphangioleiomyomatosis (LAM) is a multisystem disease affecting primarily women, characterised in the lung by proliferation of LAM cells, abnormal smooth muscle-like cells with dysfunctional tuberous sclerosis complex genes. This dysfunction results in activation of mechanistic target of rapamycin (mTOR), leading to LAM cell proliferation. Sirolimus (rapamycin) is the only United States Food and Drug Administration-approved treatment for pulmonary LAM, resulting in decreased LAM cell growth/size and stabilised lung function [1].