Therapeutic Strategies for LAM
LAM PublicationsManagement of pneumothrox in LAM
Perspectives on pneumothorx in patients with LAM
While there is currently no cure or treatment for LAM research has led to major progress in understanding and diagnosing LAM, including the identification of the genetic basis of the disease. These research efforts have prompted subsequent studies, including the first large-scale controlled clinical trial for women with LAM.
The Multicenter International LAM Efficacy of Sirolimus (MILES) Trial will test rapamycin (sirolimus), which is currently approved to prevent the immune system from rejecting kidney transplants. Learn more about this study or help enroll patients.
In early stages of the disease, most patients can go about their daily activities, but as the disease progresses, patients may have very limited mobility, require oxygen and, as a last resort, require a highly risky lung transplant. Current therapeutic approaches to LAM are primarily palliative and response to treatment varies from patient to patient.
Therapeutic strategies may include:
Hormone Therapy
Because LAM is thought to be hormone dependent, treatment strategies aimed at blocking estrogen are used, although there are no clinical studies showing that these strategies are effective. Estrogen-containing contraceptives or hormone replacement should be discontinued. For patients with progressive decline in lung function, progestin therapies (both oral and intramuscular) or gonadotropin releasing hormone (GnRH) agonists may be recommended.
Progestins
Doses of oral progestin that are sufficient to suppress serum estrogen production (e.g., Agestyn) are suggested over the use of increased doses of intramuscular progesterone that have been discussed in literature (e.g., Depoprovera). Progestins can cause fluid retention and mood swings.GnRH Agonists
GnRH agonists (e.g. Lupron) have been used in patients with LAM, but benefits are unproven and induction of early menopause is distressing and morbid in young women.Oophorectomy
Oophorectomy is no longer recommended in LAM patients. The procedure is too invasive without clear evidence of efficacy. There is also an increased risk of bone and heart disease.Pregnancy
Patients should be advised that pregnancy has been reported to result in exacerbations of LAM and pneumothorax. However, the risk of pregnancy in LAM has not been rigorously studied. Physicians and patients should discuss the risks of pregnancy carefully and decisions should be made on an individual basis.
Oxygen Therapy
Oxygen therapy may become necessary as the disease continues to worsen and lung function is impaired. Based on extrapolation from the COPD populations, the use of oxygen may prolong life in hypoxic patients with LAM. Oxygen should be administered to maintain oxyhemoglobin saturations of greater than 90 percent with rest, exercise and sleep.
Pleurodesis
Pleural disease should be aggressively managed. More than 65 percent of patients with LAM develop pneumothorax and the average number of pneumothoraces per LAM patient is 3.5. The use of a pleural symphysis procedure is recommended on the first pneumothorax, given the more than 70 percent chance of recurrence.
Chemical pleurodesis (preferably with talc), mechanical abrasion, talc poudrage and pleurectomy have all been effective in patients with LAM. Chyle does not cause pleural inflammation or fibrosis, and small chylous effusions most often require no intervention once the diagnosis is made. Shortness of breath may mandate drainage, and in some cases this may need to be repeated. Pleural effusion may be required to prevent nutritional and lymphocyte deficiencies that can result from repeated taps or persistent drainage. Chemical pleurodesis is an effective alternative for chylothorax from other etiologies.
Removal of Angiomyolipomas
Although most angiomyolipomas are asymptomatic, larger tumors (>4cm) may cause pain and bleeding.
Lung Transplantation
Referral for lung transplantation should be considered as airflow approaches 30 percent. Patients may be eligible based on other factors that profoundly affect quality of life, such as disabling dyspnea or problems maintaining oxygen saturation with lesser degrees of airflow obstruction. Survival in patients with LAM is similar to that for other lung diseases.
Bilateral lung transplantation produces slightly better functional outcomes in other obstructive lung diseases, but is not always feasible due to the limited availability of organs and urgency of the need for transplant. Recent evidence of recurrent LAM after lung transplantation suggests a metastatic mechanism for the disease.
Other Considerations
Patients with airflow obstruction should have a trial of bronchodilators and should be given influenza vaccination. Patients should be encouraged to adopt a healthy lifestyle, which includes:
- eating a healthy diet
- being as physically active as possible, as advised by your health care provider
- getting plenty of rest
- not smoking and avoiding exposure to tobacco smoke
Pulmonary rehabilitation seems to be particularly rewarding in this young, motivated population, but no studies document improvements in exercise tolerance.
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