Kristen D Starbuck1,2, Richard D Drake3, G Thomas Budd4, Peter G Rose3. 1. Department of Obstetrics and Gynecology, Cleveland Clinic, Cleveland, OH, U.S.A. Kstarbuck79@gmail.com. 2. Department of Obstetrics and Gynecology, Metrohealth Medical Center, Cleveland OH, U.S.A. 3. Division of Gynecologic Oncology, Cleveland Clinic, Cleveland, OH, U.S.A. 4. Department of Hematology and Oncology, Cleveland Clinic, Cleveland, OH, U.S.A.
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. CONCLUSION: 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. Copyright
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. CONCLUSION: 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. Copyright
Authors: Sarah Chiang; Varshini Vasudevaraja; Jonathan Serrano; Colin J R Stewart; Esther Oliva; Amir Momeni-Boroujeni; Achim A Jungbluth; Arnaud Da Cruz Paula; Edaise M da Silva; Britta Weigelt; Kay J Park; Robert A Soslow; Rajmohan Murali; Lora H Ellenson; Ryma Benayed; Marc Ladanyi; Nadeem R Abu-Rustum; Mark A Dickson; Seth Cohen; Carol Aghajanian; Martee L Hensley; Cheng-Han Lee; Matija Snuderl; Jason A Konner Journal: Mod Pathol Date: 2021-09-24 Impact factor: 8.209
Authors: K Utpatel; D F Calvisi; G Köhler; T Kühnel; A Niesel; N Verloh; M Vogelhuber; R Neu; N Hosten; H-U Schildhaus; W Dietmaier; M Evert Journal: Pathologe Date: 2020-06 Impact factor: 1.011