AUTHOR=Xu Jiaming , Gong Xiao-Lei , Wu Huanwen , Zhao Lin 

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

JOURNAL=Frontiers in Oncology

VOLUME=Volume 10 - 2020

YEAR=2020

URL=https://www.frontiersin.org/journals/oncology/articles/10.3389/fonc.2020.582087

DOI=10.3389/fonc.2020.582087

ISSN=2234-943X

ABSTRACT=Perivascular epithelioid cell tumors (PEComas) are rare mesenchymal tumors. Unresectable malignant PEComas with TFE-3 rearrangement have no recommended therapy to date. Here, we report the first case of malignant gastrointestinal PEComa with TFE3 rearrangement which has a response to the targeted therapy of an anti-VEGFR tyrosine kinase inhibitor (TKI), apatinb. A 31-year-old female was diagnosed with malignant gastrointestinal PEComa with TFE3 arrangement 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 TFE3-associated malignant PEComas.