Interstitial lung disease: Edited by Demosthenes BourosCurrent management of lymphangioleiomyomatosisTaillé, Camille; Borie, Raphaël; Crestani, BrunoAuthor Information Service de Pneumologie et Centre de Compétence des Maladies Pulmonaires Rares, Assistance Publique-Hôpitaux de Paris, Hôpital Bichat, Université Paris Diderot, Paris, France Correspondence to Professor Bruno Crestani, Service de Pneumologie, Hôpital Bichat, 46 rue Henri Huchard, 75877 Paris cedex 18, Paris, FranceTel: +33 1 40 25 68 00; fax: +33 1 40 25 88 18; e-mail: [email protected] Current Opinion in Pulmonary Medicine: September 2011 - Volume 17 - Issue 5 - p 374-378 doi: 10.1097/MCP.0b013e328349ac8c Buy Metrics Abstract Purpose of review Lymphangioleiomyomatosis (LAM) is a rare but devastating disease, leading to chronic respiratory failure. Considerable progress for comprehension of the disease has been made when mutations of the tuberous sclerosis genes TSC1 and TSC2, were discovered in LAM cells. Therapeutic consequences of these studies are important, leading to clinical trials with sirolimus for LAM. Recent findings In two studies, angiomyolipoma size decreased by 26–50% after 12 months of sirolimus treatment. In a recent 12 months controlled trial involving 89 patients with pulmonary LAM, sirolimus stopped lung function decline and improved quality of life and performance score. The protective effect of sirolimus was lost after treatment discontinuation, with a parallel lung function decline in both groups, similar to the increase in angiomyolipoma size. Sirolimus is associated with an excess of adverse events. Summary Sirolimus represents an important drug for LAM that should be proposed to patients with a rapid alteration of lung function or with a significant clinical impairment, after individual evaluation of the risk/benefit ratio. Sirolimus seems to have a sharper effect on the reduction of abdominal masses than on lung cysts. Tolerance and safety concerns are serious limits to the long-term treatment of patients with sirolimus. © 2011 Lippincott Williams & Wilkins, Inc.