Lymphangioleiomyomatosis Medication

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

Medication Summary

The goals of pharmacotherapy for lymphangioleiomyomatosis (LAM) are to reduce morbidity and to prevent complications. However, new drug therapies target the underlying disease.

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Immunosuppressant agents

Class Summary

Immunosuppressants may inhibit T-lymphocyte activation and proliferation that come about from antigenic and cytokine stimulation.

Sirolimus (Rapamune)

LAM involves lung tissue infiltration with smooth muscle-like cells that harbor inactivating mutations of the tuberous sclerosis complex (TSC) gene (LAM cells). Loss of TSC gene function activates the (mechanistic target of rapamycin (mTOR) signaling pathway, resulting in cellular proliferation and release of lymphangiogenic growth factors. Sirolimus inhibits the activated mTOR pathway and thus the proliferation of LAM cells. It received FDA approval for treatment of LAM in May 2015. 

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Antimalarials, Aminoquinoline

Class Summary

Aminoquinolines may inhibit prostaglandin effects.

Chloroquine Phosphate (Aralen)

Chloroquine is an autophagy inhibitor that appears to induce the death of TSC2-deficient cells when given in combination with sirolimus and is being tested in an ongoing clinical trial.

Anti-inflammatory activity results from lymphocyte transformation suppression. It inhibits chemotaxis of eosinophils and locomotion of neutrophils and impairs complement-dependent antigen-antibody reactions. Chloroquine may also have photoprotective effect.

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Antineoplastics, Aromatase Inhibitor

Class Summary

Aromatase inhibitors can cause a significant reduction in plasma estrogen.

Letrozole (Femara)

Letrozole is an aromatase inhibitor with antiestrogenic effects that is being tested in LAM.

It is a nonsteroidal competitive inhibitor of the aromatase enzyme system. It inhibits the conversion of androgens to estrogens. Letrozole selectively inhibits gonadal steroidogenesis but has no significant effect on adrenal mineralocorticoid or glucocorticoid synthesis.

Patients treated with letrozole do not require glucocorticoid or mineralocorticoid replacement therapy.

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