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
• ISSN: 0028-4793; 1533-4406; 1533-4406
• DOI: 10.1056/NEJMoa1100391

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 systemic disease that is associated with cystic destruction of the lung, chylous pleural effusions, and abdominal tumors such as renal angiomyolipomas. 1 , 2 LAM affects women almost exclusively and occurs sporadically, developing in about 5 persons per 1 million; it also affects 30 to 40% of women with tuberous sclerosis complex (TSC). Lung function, measured as the forced expiratory volume in 1 second (FEV 1 ), declines at the rate of 75 to 118 ml per year 3 – 5 ; clinically important respiratory impairment, recurrent pneumothoraxes, and hypoxemia develop in most patients within a decade after the . . .