Lymphangioleiomyomatosis: current and future

Lymphangioleiomyomatosis is a rare slowly progressive lung disease that affects almost exclusively young women of reproductive age. It occurs sporadically or in association with Tuberous Sclerosis Complex. LAM is characterized by cystic remodeling of the lung parenchyma, due to proliferation of abnormal smooth muscle-like LAM cells and presence of extra pulmonary manifestations such as lymphadenopathy, angiomyolipomas and abdominal lymphangioleiomyomas. The most common clinical manifestations are progressive dyspnea on exertion, pneumothorax and chylous effusions. Currently there is no curative treatment for the disease, but the ongoing study of the genetic and molecular pathways implicated in the pathogenesis of the disease could lead to targeted therapy.