title: Neoadjuvant or adjuvant sirolimus for malignant metastatic or locally advanced perivascular epithelioid cell tumors: two case reports.
abstract: Perivascular epithelioid cell tumors (PEComas) are very rare mesenchymal tumors, characterized by the presence of perivascular epithelioid cells. Despite their often benign nature, malignant variants with a locally aggressive growth pattern and even distant metastases are known. We describe two cases of malignant PEComas. The first patient had an extensive peritoneal spread and a history of multiple resections, and received the mechanistic target of rapamycin inhibitor sirolimus in a postoperative setting as maintenance therapy. The second patient presented with locally advanced disease in the iliac fossa and was treated with sirolimus in a neoadjuvant setting and achieved complete remission. Both patients have been under treatment for 18 and 52 months, respectively, and are currently in complete remission. These two cases indicate that mechanistic target of rapamycin inhibition for malignant PEComas could be a safe and successful treatment strategy in a neoadjuvant setting with an acceptable toxicity profile.
authors.affiliation: Departments of aInternal Medicine III bGeneral, Visceral, Transplantation, Vascular, and Thoracic Surgery cOrthopaedic Surgery, Orthopaedic Oncology dClinical Radiology eInstitute of Pathology fCenter for Bone and Soft Tissue Sarcoma (SarKUM) gComprehensive Cancer Center, Ludwig-Maximilians-University of Munich, Munich, Germany.
authors.forename: Christiane
authors.initials: C
authors.lastname: Batereau
authors.forename: Thomas
authors.initials: T
authors.lastname: Knösel
authors.forename: Martin
authors.initials: M
authors.lastname: Angele
authors.forename: Hans Roland
authors.initials: HR
authors.lastname: Dürr
authors.forename: Melvin
authors.initials: M
authors.lastname: D'Anastasi
authors.forename: Eric
authors.initials: E
authors.lastname: Kampmann
authors.forename: Bernhard
authors.initials: B
authors.lastname: Ismann
authors.forename: Veit
authors.initials: V
authors.lastname: Bücklein
authors.forename: Lars H
authors.initials: LH
authors.lastname: Lindner
mesh_terms: D000328:Adult; D000903:Antibiotics, Antineoplastic / Q000627:therapeutic use*; D017024:Chemotherapy, Adjuvant; D005260:Female; D006801:Humans; D060046:Maintenance Chemotherapy; D008875:Middle Aged; D020360:Neoadjuvant Therapy; D009362:Neoplasm Metastasis; D054973:Perivascular Epithelioid Cell Neoplasms / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery; D020123:Sirolimus / Q000627:therapeutic use*; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors
chemical_list: D000903:Antibiotics, Antineoplastic; C546842:MTOR protein, human; D058570:TOR Serine-Threonine Kinases; D020123:Sirolimus