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Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis
Open accessAbstract
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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“Although exposure to mTOR inhibitors in the immediate post-transplant period has been associated with delayed bronchial anastomotic healing or anastomotic dehiscence, mTOR inhibitors are now successfully continued up until the time transplant in many centers without complications.(305, 315, 320)”
From Consensus document for the selection of lung transplant candidates: An update from the International Society for Heart and Lung Transplantation. (2021) · cites this paper as [315]
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“Although exposure to mTOR inhibitors in the immediate post-transplant period has been associated with delayed bronchial anastomotic healing or anastomotic dehiscence, mTOR inhibitors are now successfully continued up until the time transplant in many centers without complications.(305, 315, 320)”
From Consensus document for the selection of lung transplant candidates: An update from the International Society for Heart and Lung Transplantation. (2021) · cites this paper as [315]
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Self-citation
Metadata
- 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
- 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
- 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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