Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis

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...

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Vydané v:The New England journal of medicine Ročník 364; číslo 17; s. 1595 - 1606
Hlavní autori: 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
Médium: Journal Article
Jazyk:English
Vydavateľské údaje: United States Massachusetts Medical Society 28.04.2011
Predmet:
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
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Shrnutí: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 . . .
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The members of the Rare Lung Diseases Consortium and the Multicenter International Lymphangioleiomyomatosis Efficacy and Safety of Sirolimus (MILES) trial group are listed in the Supplementary Appendix, available at NEJM.org.
ISSN:0028-4793
1533-4406
1533-4406
DOI:10.1056/NEJMoa1100391