Cited by 1,099Tuberous Sclerosis Complex ResearchVascular Malformations and HemangiomasPulmonary Hypertension Research and Treatments

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Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis

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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.·2011·New England Journal of Medicine

DOI 10.1056/nejmoa1100391PMC PMC3118601

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Abstract

BACKGROUND: Lymphangioleiomyomatosis (LAM) is a progressive, cystic lung disease in women; it is associated with inappropriate activation of mammalian target of rapamycin (mTOR) signaling, which regulates cellular growth and lymphangiogenesis. Sirolimus (also called rapamycin) inhibits mTOR and has shown promise in phase 1-2 trials involving patients with LAM. METHODS: We conducted a two-stage trial of sirolimus involving 89 patients with LAM who had moderate lung impairment--a 12-month randomized, double-blind comparison of sirolimus with placebo, followed by a 12-month observation period. The primary end point was the difference between the groups in the rate of change (slope) in forced expiratory volume in 1 second (FEV(1)). RESULTS: During the treatment period, the FEV(1) slope was -12±2 ml per month in the placebo group (43 patients) and 1±2 ml per month in the sirolimus group (46 patients) (P<0.001). The absolute between-group difference in the mean change in FEV(1) during the treatment period was 153 ml, or approximately 11% of the mean FEV(1) at enrollment. As compared with the placebo group, the sirolimus group had improvement from baseline to 12 months in measures of forced vital capacity, functional residual capacity, serum vascular endothelial growth factor D (VEGF-D), and quality of life and functional performance. There was no significant between-group difference in this interval in the change in 6-minute walk distance or diffusing capacity of the lung for carbon monoxide. After discontinuation of sirolimus, the decline in lung function resumed in the sirolimus group and paralleled that in the placebo group. Adverse events were more common with sirolimus, but the frequency of serious adverse events did not differ significantly between the groups. CONCLUSIONS: In patients with LAM, sirolimus stabilized lung function, reduced serum VEGF-D levels, and was associated with a reduction in symptoms and improvement in quality of life. Therapy with sirolimus may be useful in selected patients with LAM. (Funded by the National Institutes of Health and others; MILES ClinicalTrials.gov number, NCT00414648.).

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Authors: McCormack, F. X., Inoue, Y., Moss, J., Singer, L. G., Strange, C., Nakata, K., Barker, A. F., Chapman, J. T., Brantly, M. L., Stocks, J. M., Brown, K. K., Lynch, J. P., Goldberg, H. J., Young, L. R., Kinder, B. W., Downey, G. P., Sullivan, E. J., Colby, T. V., McKay, R. T., Cohen, M. M., Korbee, L., Taveira-DaSilva, A. M., Lee, H.-S., Krischer, J. P., & Trapnell, B. C.
Affiliations: University of Cincinnati (US)
National Hospital Organization (JP)
National Institutes of Health (US)
University of Toronto (CA)
Medical University of South Carolina (US)
Niigata University Medical and Dental Hospital (JP)
Oregon Health & Science University (US)
Cleveland Clinic (US)
University of Florida (US)
The University of Texas Health Science Center at Tyler (US)
National Jewish Health (US)
Cincinnati Children's Hospital Medical Center (US)
Harvard University (US)
United Therapeutics (United States) (US)
Mayo Clinic (US)
University of South Florida (US)
ORCID: McCormack, Francis X.
Inoue, Yoshikazu
Moss, Joel
Singer, Lianne G.
Strange, Charlie
Nakata, Koh
Brantly, Mark L.
Brown, Kevin K.
Goldberg, Hilary J.
Young, Lisa R.
Downey, Gregory P.
Colby, Thomas V.
Lee, Hye-Seung
Krischer, Jeffrey P.
Trapnell, Bruce C.
Venue: New England Journal of Medicine
Publisher: Massachusetts Medical Society
Published: 2011-04-28
Volume: 364
Issue: 17
Pages: 1595-1606
ISSN: 0028-4793 (ISSN-L)
1533-4406
DOI: 10.1056/nejmoa1100391
PMC: PMC3118601
OpenAlex: W2147028601
Semantic Scholar: 0af4a52c455e626ae3c70aa2ba2d64208b19a305
Cited by: 1,099
References: 27
Citations by year: 2026: 38 · 2025: 54 · 2024: 77 · 2023: 70 · 2022: 80 · 2021: 70 · 2020: 72 · 2019: 78 · 2018: 77 · 2017: 65 · 2016: 78 · 2015: 87 · 2014: 71 · 2013: 81 · 2012: 68
FWCI: 54.96
Type: journal article
Language: en
Open access: bronze
doi.org
Locations: New England Journal of Medicine (publishedVersion)
PubMed (publishedVersion)
Europe PMC (PubMed Central) (acceptedVersion)
PubMed Central (acceptedVersion)
Funders: Tuberous Sclerosis Alliance
Pfizer
LAM Foundation
Cincinnati Children's Hospital Medical Center
National Institutes of Health (1ul1rr026314-01, 1UL1RR026314)
U.S. Food and Drug Administration (FD003362, R01 FD003362)
Canadian Institutes of Health Research
National Heart, Lung, and Blood Institute (R01HL085453, ZIAHL002541)
National Center for Research Resources (1UL1 RR-026314-01, U54RR019259, UL1-RR026314, U54RR019498, RR019498, RR019259)
NHLBI Division of Intramural Research
Topics: Health Sciences › Medicine › Genetics (clinical) › Tuberous Sclerosis Complex Research
Health Sciences › Medicine › Surgery › Vascular Malformations and Hemangiomas
Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Pulmonary Hypertension Research and Treatments
Keywords: lymphangioleiomyomatosis, rapamycin, mTOR signaling, lung function, FEV1, VEGF-D, quality of life, adverse events, drug discontinuation, cystic lung disease
MeSH terms: Adult, Double-Blind Method, Female, Forced Expiratory Volume, Humans, Middle Aged, Quality of Life, Vital Capacity, Lymphangioleiomyomatosis, Observation, Sirolimus, Medication Adherence, Intention to Treat Analysis, TOR Serine-Threonine Kinases

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Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis

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