SUCCESSFUL TREATMENT OF PULMONARY AND LYMPHATIC MANIFESTATIONS OF LYMPHANGIOLEIOMYOMATOSIS WITH SIROLIMUS

Abstract

Lymphangioleiomyomatosis (LAM) is arare, progressive, diffuse cystic lung diseasepredominantly affecting women of childbearing age. Recently treatment with sirolimuswas shown to stabilize lung function declineand improve quality of life in patients withLAM. We treated three premenopausalwomen suffering from LAM manifesting asdiffuse cystic lung disease, chylous effusions,and lymphangioleioyomas with sirolimus (1-3mg a day; sirolimus trough levels 2.9-8.5ng/ml). All three patients had a remarkableresponse to sirolimus, with resolution ofeffusions, improvement in lung function andshrinking of abdominal lymphangioleiomyomas.Our case series further complementsthe literature in that sirolimus is a safe andeffective treatment for LAM and its lymphaticmanifestations.