Sirolimus Treatment for Pulmonary Lymphangioleiomyomatosis

Letters1 May 2007Sirolimus Treatment for Pulmonary LymphangioleiomyomatosisCamille Taillé, MD, PhD, Marie-Pierre Debray, MD, and Bruno Crestani, PhDCamille Taillé, MD, PhDFrom Hôpital Bichat–Claude Bernard and Hôpitaux de Paris, Paris 75018, France., Marie-Pierre Debray, MDFrom Hôpital Bichat–Claude Bernard and Hôpitaux de Paris, Paris 75018, France., and Bruno Crestani, PhDFrom Hôpital Bichat–Claude Bernard and Hôpitaux de Paris, Paris 75018, France.Author, Article, and Disclosure Informationhttps://doi.org/10.7326/0003-4819-146-9-200705010-00022 SectionsAboutFull TextPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissions ShareFacebookTwitterLinkedInRedditEmail Background: Lymphangioleiomyomatosis (LAM) is a rare disease characterized by abnormal proliferation of smooth-muscle cells within the lung (1) that are responsible for the cystic destruction of lung parenchyma, leading to chronic respiratory failure (2). No effective treatment, except for lung transplantation, is available. Abdominal tumors (renal angiomyolipomas, lymphangioleiomyomas, and enlarged lymph nodes) are frequently associated with lung involvement. Recently, loss-of-function mutations in the tumor suppressor genes tuberous sclerosis 1 and 2 (TSC1 and TSC2, respectively) have been described in LAM (3). In vitro studies on LAM smooth-muscle cells have shown that mutation on the TSC1–TSC2 complex dysregulates the activation of ...References1. Finlay G. The LAM cell: what is it, where does it come from, and why does it grow? [Editorial]. Am J Physiol Lung Cell Mol Physiol. 2004;286:L690-3. [PMID: 15003933] CrossrefMedlineGoogle Scholar2. Sullivan EJ. Lymphangioleiomyomatosis: a review. Chest. 1998;114:1689-703. [PMID: 9872207] CrossrefMedlineGoogle Scholar3. Strizheva GD, Carsillo T, Kruger WD, Sullivan EJ, Ryu JH, Henske EP. The spectrum of mutations in TSC1 and TSC2 in women with tuberous sclerosis and lymphangiomyomatosis. Am J Respir Crit Care Med. 2001;163:253-8. [PMID: 11208653] CrossrefMedlineGoogle Scholar4. Goncharova EA, Goncharov DA, Eszterhas A, Hunter DS, Glassberg MK, Yeung RS, et al. Tuberin regulates p70 S6 kinase activation and ribosomal protein S6 phosphorylation. A role for the TSC2 tumor suppressor gene in pulmonary lymphangioleiomyomatosis (LAM). J Biol Chem. 2002;277:30958-67. [PMID: 12045200] CrossrefMedlineGoogle Scholar5. Goncharova EA, Goncharov DA, Spaits M, Noonan DJ, Talovskaya E, Eszterhas A, et al. Abnormal growth of smooth muscle-like cells in lymphangioleiomyomatosis: role for tumor suppressor TSC2. Am J Respir Cell Mol Biol. 2006;34:561-72. [PMID: 16424383] CrossrefMedlineGoogle Scholar Author, Article, and Disclosure InformationAffiliations: From Hôpital Bichat–Claude Bernard and Hôpitaux de Paris, Paris 75018, France.Disclosures: None disclosed. 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