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Genetic and Rare Diseases Information Center (GARD)


Other Names for this Disease
  • LAM
  • Lymphangio-myomatosis
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How is lymphangioleiomyomatosis (LAM) treated?

While there is currently no cure for LAM, research has led to major progress in the treatment of symptoms, and diagnosing and understanding LAM. There are a number of treatments that may relieve symptoms or prevent complications from the disease. Treatments vary from patient to patient, depending on the severity of the disease. No all patients have the same response to treatment.[1][2]

  • Medicines (e.g., diuretics, hormone therapy, bronchodilators that relax the muscles around the airways)
  • Oxygen therapy - as lung capacity declines, supplemental or full-time oxygen therapy may become necessary
  • Sirolimus therapy - Rapamycin (sirolimus) blocks the pathway that is affected in LAM cells, which helps stop their uncontrolled growth. The use of sirolimus therapy may be considered in patients with moderate to severe LAM, or those with progressive disease.
  • Procedures to remove air or fluid from the chest or abdominal cavities and prevent it from building up again
  • Procedures to remove angiomyolipoma (AML), or benign kidney tumors
  • Lung transplantation - a procedure to replace one or both lungs, this should be considered as a last resort due to the risk of serious adverse reactions, including major bleeding, pneumonia, pulmonary edema, long-term infections, and possibly painful scarring.
  • Since LAM occurs almost exclusively in women of reproductive age, researchers believe the hormone estrogen might be involved in the abnormal muscle cell growth that characterizes the disease. Although there is no direct evidence that there is a relationship between estrogen and LAM, the treatment of LAM has focused on reducing the production or effects of estrogen. This could include estrogen or other hormone suppressing drugs. Additionally, doctors believe pregnancy may accelerate the progression of LAM. Women with LAM are urged to speak with a health care professional before getting pregnant.[2]

    Last updated: 7/16/2013

    1. How Is LAM Treated?. National Heart Lung and Blood Institute (NHLBI). December 26, 2013; Accessed 7/21/2014.
    2. Treating LAM. The LAM Foundation. 2014; Accessed 7/21/2014.

    Clinical Trials & Research for this Disease

    • lists trials that are studying or have studied Lymphangioleiomyomatosis. Click on the link to go to to read descriptions of these studies.
    • The Rare Lung Diseases Consortium: Molecular Pathway-Driven Diagnostics and Therapeutics for Rare Lung Diseases is an integrated group of academic medical centers, patient support organizations, and clinical research resources dedicated to conducting clinical research involving pulmonary alveolar proteinosis, Hermansky-Pudlak Syndrome, and Lymphangioleiomyomatosis. A pilot project program supports research into other rare lung diseases that complement the main research projects.

    Medical Products

    The medication(s) listed in the table(s) below have been approved by the Food and Drug Administration (FDA) for treatment of this condition. The FDA Office of Orphan Products Development designates "orphan products" for those that treat rare diseases affecting fewer than 200,000 Americans. The table(s) below may not be an exhaustive list of drugs or products used to treat this condition. There may be other products available that are not considered orphan products. To search for all FDA approved drugs, visit Drugs@FDA. You can find orphan products used to treat other conditions by searching the Orphan Drug Product Designation database.

    Generic Name Sirolimus
    Trade Name
    (Manufacturer Name)
    (Pfizer, Inc.)
    The FDA has approved this product to be used in this manner.
    Treatment of lymphangioleiomyomatosis
    More Information about this product Drug Information Portal
    Medline Plus Health Information

    Other Names for this Disease
    • LAM
    • Lymphangio-myomatosis
    See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.