title: SUCCESSFUL TREATMENT OF PULMONARY AND LYMPHATIC MANIFESTATIONS OF LYMPHANGIOLEIOMYOMATOSIS WITH SIROLIMUS.
abstract: Lymphangioleiomyomatosis (LAM) is a rare, progressive, diffuse cystic lung disease predominantly affecting women of child bearing age. Recently treatment with sirolimus was shown to stabilize lung function decline and improve quality of life in patients with LAM. We treated three premenopausal women suffering from LAM manifesting as diffuse cystic lung disease, chylous effusions, and lymphangioleioyomas with sirolimus (1-3 mg a day; sirolimus trough levels 2.9-8.5 ng/ml). All three patients had a remarkable response to sirolimus, with resolution of effusions, improvement in lung function and shrinking of abdominal lymphangioleiomyomas. Our case series further complements the literature in that sirolimus is a safe and effective treatment for LAM and its lymphatic manifestations.
authors.forename: A
authors.initials: A
authors.lastname: Hecimovic
authors.forename: M
authors.initials: M
authors.lastname: Jakopovic
authors.forename: G
authors.initials: G
authors.lastname: Pavlisa
authors.forename: M
authors.initials: M
authors.lastname: Jankovic
authors.forename: A
authors.initials: A
authors.lastname: Vukic-Dugac
authors.forename: G
authors.initials: G
authors.lastname: Redzepi
authors.forename: L
authors.initials: L
authors.lastname: Brcic
authors.forename: M
authors.initials: M
authors.lastname: Samarzija
authors.forename: N
authors.initials: N
authors.lastname: Gupta
mesh_terms: D000328:Adult; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D014408:Biomarkers, Tumor / Q000032:analysis; D001706:Biopsy; D005260:Female; D006801:Humans; D007150:Immunohistochemistry; D008175:Lung Neoplasms / Q000737:chemistry / Q000175:diagnosis / Q000188:drug therapy*; D018192:Lymphangioleiomyomatosis / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism; D012129:Respiratory Function Tests; D012186:Retroperitoneal Neoplasms / Q000737:chemistry / Q000175:diagnosis / Q000188:drug therapy*; D020123:Sirolimus / Q000627:therapeutic use*; D014057:Tomography, X-Ray Computed; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D014408:Biomarkers, Tumor; D020123:Sirolimus