title: Treatment of Advanced Malignant Uterine Perivascular Epithelioid Cell Tumor with mTOR Inhibitors: Single-institution Experience and Review of the Literature.
abstract: Uterine perivascular epithelioid cell tumors (PEComas) are rare mesenchymal tumors. Many have malignant behavior, and no successful treatment strategy has been established. Identification of mutations in the tuberous sclerosis 1 (TSC1) and TSC2 genes producing constitutive activation of the mammalian target of rapamycin (mTOR) pathway presents an opportunity for targeted therapy. Patients with advanced malignant uterine PEComa treated with mTOR inhibitors were identified and records were retrospectively reviewed for treatment response based on radiographic assessment. Three patients with advanced uterine PEComas underwent debulking surgery followed by mTOR inhibitor therapy; two had a complete response to therapy and disease in one patient progressed.
  
  
  
  Given the absence of effective therapies for malignant uterine PEComas, targeting the mTOR pathway is a logical strategy to pursue given the known pathobiology involving the Tuberous Sclerosis complex. Treatment of malignant uterine PEComas with mTOR inhibitors was effective in two out of three patients after surgical resection, with durable response.
authors: Department of Obstetrics and Gynecology, Cleveland Clinic, Cleveland, OH, U.S.A. Kstarbuck79@gmail.com.|Department of Obstetrics and Gynecology, Metrohealth Medical Center, Cleveland OH, U.S.A.
authors: Kristen D
authors: KD
authors: Starbuck
authors: Division of Gynecologic Oncology, Cleveland Clinic, Cleveland, OH, U.S.A.
authors: Richard D
authors: RD
authors: Drake
authors: Department of Hematology and Oncology, Cleveland Clinic, Cleveland, OH, U.S.A.
authors: G Thomas
authors: GT
authors: Budd
authors: Division of Gynecologic Oncology, Cleveland Clinic, Cleveland, OH, U.S.A.
authors: Peter G
authors: PG
authors: Rose
mesh_terms: D000328:Adult; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D008875:Middle Aged; D054973:Perivascular Epithelioid Cell Neoplasms / Q000188:drug therapy*; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors*; D016896:Treatment Outcome; D014594:Uterine Neoplasms / Q000188:drug therapy*
chemical_list: D000970:Antineoplastic Agents; C546842:MTOR protein, human; D058570:TOR Serine-Threonine Kinases
keywords: PEComa; mTOR inhibitor; perivascular epithelioid cell tumor; sirolimus; temsirolimus