Role of Chemotherapy, VEGFR Inhibitors, and mTOR Inhibitors in Advanced Perivascular Epithelioid Cell Tumors (PEComas).

Role of Chemotherapy, VEGFR Inhibitors, and mTOR Inhibitors in Advanced Perivascular Epithelioid Cell Tumors (PEComas).

Publication date: Sep 01, 2019

Perivascular epitheliod cell tumors (PEComas) are rare mesenchymal neoplasms for which the role of systemic treatments is not established as there are no published prospective clinical trials or sufficiently large retrospective case series. The aim of this study is to clarify the activity of conventional chemotherapy and biological agents in advanced/metastatic PEComas.

This was an observational, retrospective, international study that included patients with advanced/metastatic PEComa treated with systemic therapy at 5 European sarcoma reference centers and within the Italian Rare Cancer Network. Survival analyses were performed using the Kaplan-Meier method and the Cox hazards regression models.

A total of 53 patients were included. Cytotoxic chemotherapy regimens were active only in a small proportion of PEComas. Gemcitabine-based regimens [objective response rate (ORR): 20%, median progression-free survival (PFS): 3.4 months] seemed to have the same activity of anthracycline-based regimens (ORR: 13%, median PFS: 3.2 months). Antiangiogenic agents resulted in disease stabilization in some patients, with a number having density changes/tissue response on imaging, with an ORR of 8.3% and a median PFS of 5.4 months. mTOR inhibitors were the most active agents, with an ORR of 41% and a median PFS of 9 months.

Our study provides data for the selection of systemic therapy in patients with advanced/metastatic PEComa: mTOR inhibitors are the most active agents. Antiangiogenics and chemotherapy with gemcitabine-based regimens or anthracycline-based regimens are options in further line, but with a lower response rate and PFS.

Sanfilippo, R., Jones, R.L., Blay, J.Y., Le Cesne, A., Provenzano, S., Antoniou, G., Mir, O., Fuc`a, G., Fumagalli, E., Bertulli, R., Stacchiotti, S., Brahmi, M., Grosso, F., Dufresne, A., Hindi, N., Sbaraglia, M., Gronchi, A., Collini, P., Dei Tos, A.P., and Casali, P.G. Role of Chemotherapy, VEGFR Inhibitors, and mTOR Inhibitors in Advanced Perivascular Epithelioid Cell Tumors (PEComas). 24043. 2019 Clin Cancer Res (25):17.

Concepts Keywords
Anthracycline Progression-free survival
Chemotherapy Chemotherapy
Chemotherapy Regimens Oncology
Clinical Trials Antineoplastic drugs
Gemcitabine Cancer treatments
Italian Cancer
Mesenchymal Clinical medicine
Metastatic Medicine
MTOR Chemotherapy
Neoplasms Tumors
Regression Neoplasms
Sarcoma Imaging
Systemic Therapy Chemotherapy
VEGFR Inhibitors Evofosfamide
Mantle cell lymphoma
MTOR inhibitors

Semantics

Type Source Name
drug DRUGBANK Gemcitabine
gene UNIPROT CPOX
disease DOID Cancer
disease DOID sarcoma
disease MESH sarcoma
disease DOID PEComa
gene UNIPROT DNMT1
gene UNIPROT CD69
gene UNIPROT LARGE1
disease MESH tumors
disease MESH Perivascular Epithelioid Cell Tumors
gene UNIPROT MTOR

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