Case Report: Gastrointestinal PEComa With TFE3 Rearrangement Treated With Anti-VEGFR TKI Apatinib

Abstract

Perivascular epithelioid cell tumors (PEComas) are rare mesenchymal tumors. Unresectable malignant PEComas with <i>TFE3</i> rearrangement have no recommended therapy to date. Here, we report the first case of malignant gastrointestinal PEComa with <i>TFE3</i> rearrangement which has a response to the targeted therapy of an anti-VEGFR tyrosine kinase inhibitor (TKI), apatinib. A 31-year-old female was diagnosed with malignant gastrointestinal PEComa with <i>TFE3</i> rearrangement and hepatic metastases. A resection of the giant retroperitoneal mass was performed. The patient received the anti-VEGFR TKI apatinib to treat the hepatic metastasis. The tumor remained stable during apatinib treatment and the progression-free survival (PFS) lasted about 7 months. This case suggests that targeting the VEGF/VEGFR signaling pathway may be an essential new therapeutic choice for <i>TFE3</i>-associated malignant PEComas.