While there is currently no cure for LAM, there are a number of treatments that may relieve symptoms or prevent complications from the disease.
First-Ever Clinical Treatment for LAM
Siroliumus (also known as rapamycin or Rapamune) is the first FDA approved treatment for lymphangioleiomyomatosis. It has been shown to stabilize the disease and prevent it from getting worse while also improving quality of life for patients with LAM. Sirolimus has also proved as an effective treatment for LAM patients with chylous effusions and angiomyolipomas of the kidney. Symptoms tend to reappear once the sirolimus is stopped so further studies investigating the long term efficacy of this treatment for lymphangioleiomyomatosis still need to be explored.
Patients have experienced some side effects while taking sirolimus, such as swelling of the hands, feet and ankles, acne, mouth ulcers, indigestion, diarrhea, elevation of cholesterol and triglyceride levels, hypertension and headache. It’s also important to note that sirolimus inhibits wound healing and it is often necessary to stop taking the drug for a period of time before and after elective procedures when wound healing will take place. Patients taking sirolimus are also cautioned to avoid prolonged exposure to the sun because of an increased risk of skin cancer for those taking the drug.
Available (Palliative) Treatment Options
There are several current treatments that are considered palliative. That is, they do not treat LAM, but aim to relieve symptoms or prevent additional complications from the disease. The response to treatment varies from patient to patient. Potential treatments include:
Role of Hormones
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.