Lymphangioleiomyomatosis Treatment & Management

Updated: Dec 22, 2019
  • Author: Joel Moss, MD, PhD; Chief Editor: Zab Mosenifar, MD, FACP, FCCP  more...
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Treatment

Approach Considerations

General care for patients with lymphangioleiomyomatosis (LAM) addresses the following findings [13] :

  • Pleural effusions - Consider chemical pleurodesis; surgical obliteration of the pleural space; medium-chain triglyceride (MCT [not a component of chyle]), lipid-free diet to reduce chyle flow (utility unknown)

  • Ascites - Paracentesis, MCT diet (utility unknown)

  • Airways disease and hypoxemia - Bronchodilators may be of benefit [16] ; supplemental oxygen, pulmonary rehabilitation, smoking cessation

  • Standard vaccination for respiratory infections

  • Osteoporosis - Standard surveillance and treatment; avoid exogenous estrogens [30]

​Some reports suggest worsening of disease during pregnancy. Therefore, specialists generally recommend that caution be exercised when patients consider becoming pregnant. Despite the potential hazards, patients with mild disease have had uneventful pregnancies with little deterioration in lung function.

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Medical Care

Sirolimus

In May 2015, sirolimus (Rapamune) was the first drug approved by the US FDA for LAM. It has demonstrated improvement in lung function in patients with LAM. Sirolimus also reduces the size of angiomyolipomas, lymphangioleiomyomas, and chylous effusions. The therapeutic response, however, is generally not sustained. [37, 38, 39, 40]

A clinical trial of 89 patients over 12 months showed patients taking Rapamune had a slower decline in lung function than those taking a placebo. After the drug was stopped, the decline in lung function resumed at the same rate as the placebo group. [37]

Hormone therapy

Possible options for hormonal manipulation include the following:

  • Medroxyprogesterone - Utility not known; recent case series does not support its use [41]

  • Gonadotropin-releasing hormone agonists - Utility not known; few case reports support their use

  • Tamoxifen does not appear to be effective [31] and is not recommended due to estrogen receptor agonist activity

  • Rate of decline in lung function trends to be less in postmenopausal women (eg, surgical oophorectomy, age)

Novel therapies

New experimental therapies include the following:

  • Chloroquine - Inhibitor of autophagy

  • Aromatase inhibitors - Antiestrogenic effects

Everolimus, an oral mTOR inhibitor, has been investigated as a treatment of renal angiomyolipoma associated with TSC or sporadic LAM. [42] A 4-year double-blind, placebo-controlled, phase 3 follow-up trial showed reduction in angiomyolipoma volume in 54% of patients. [19] During the extension phase of the study, 58% of patients achieved angiomyolipoma response. The most common adverse events reported were stomatitis (42%), hypercholesterolemia (30.4%), acne (25.9%), aphthous stomatitis and nasopharyngitis (each 21.4%). [18]

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Surgical Care

Management of recurrent pneumothoraces or pleural effusions may require surgical intervention; in addition, patients with AML can develop complications (eg, hemorrhage), requiring intervention. Arterial embolization is favored over surgical resection. [43]

Consider lung transplantation for patients with end-stage pulmonary disease.

The Medscape Transplantation Specialty Center may be helpful.

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Consultations

Pulmonologists can help establish the diagnosis and monitor pulmonary function. A pulmonologist can address issues such as vaccinations, oxygen therapy, and pulmonary rehabilitation. Consider patient referral to a center with expertise in lymphangioleiomyomatosis (LAM). Depending on disease severity, referral to a transplantation center may be beneficial.

AN endocrinologist and/or obstetrician-gynecologist may help address issues involved in hormonal manipulation therapy. An endocrinologist can assist with prophylaxis and treatment of osteoporosis in patients in whom exogenous estrogen is contraindicated. A urologist may assist with management of renal AML.

A dietitian may help to advise patients on medium chain triglyceride (MCT) diets for chylous ascites or pleural effusions.

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Diet

Most patients with lymphangioleiomyomatosis (LAM) do not have special dietary requirements; however, if a patient is on antiestrogen therapies or is postmenopausal and not on estrogen replacement therapy, other cardiac risk factors relevant to diet (eg, cholesterol levels) should be addressed.

Although the use of rapamycin is favored, some patients with chylous effusions or ascites may try an medium chain triglyceride (MCT) diet (see Medical Care).

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Activity

LAM is associated with reduced bone mineral density and osteoporosis. Weight-bearing exercise and strength training should be encouraged. [13]

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