Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis

• Published in: The New England journal of medicine Vol. 364; no. 17; pp. 1595 - 1606
• Main Authors: McCormack, Francis X, Inoue, Yoshikazu, Moss, Joel, Singer, Lianne G, Strange, Charlie, Nakata, Koh, Barker, Alan F, Chapman, Jeffrey T, Brantly, Mark L, Stocks, James M, Brown, Kevin K, Lynch, Joseph P, Goldberg, Hilary J, Young, Lisa R, Kinder, Brent W, Downey, Gregory P, Sullivan, Eugene J, Colby, Thomas V, McKay, Roy T, Cohen, Marsha M, Korbee, Leslie, Taveira-DaSilva, Angelo M, Lee, Hye-Seung, Krischer, Jeffrey P, Trapnell, Bruce C
• Format: Journal Article
• Language: English
• Published: United States Massachusetts Medical Society 28.04.2011
• Subjects: Adult; Double-Blind Method; Female; Forced Expiratory Volume - drug effects; Human subjects; Humans; Intention to Treat Analysis; Kinases; Lungs; Lymphangioleiomyomatosis - drug therapy; Lymphangioleiomyomatosis - physiopathology; Medication Adherence; Middle Aged; Observation; Quality of Life; Sirolimus - adverse effects; Sirolimus - blood; Sirolimus - therapeutic use; TOR Serine-Threonine Kinases - antagonists & inhibitors; Vascular endothelial growth factor; Vital Capacity - drug effects; Womens health

This trial showed that in patients with lymphangioleiomyomatosis (LAM), a progressive, cystic lung disease in women, sirolimus therapy was associated with stabilization of lung function, reduction in symptoms, and improvement in qualityof-life measures. Lymphangioleiomyomatosis (LAM) is an uncommon...