Other names: Malignant Perivascular Epithelioid Cell Neoplasm; PEComa

Malignant Perivascular Epithelioid Cell Tumors (PEComas) are a rare and aggressive form of soft-tissue sarcomas. 

PEComa is defined by the World Health Organization as 'mesenchymal tumors composed of distinctive cells that show a focal association with blood-vessel walls and usually express both melanocytic and smooth muscle markers'.

Malignant PEComas have a strong female predominance and may arise in almost any body site, including the uterus, retroperitoneum, lung, kidney, liver, genitourinary, and gastrointestinal tract.

Malignant PEComas have been shown to frequently harbor mutations in the TSC1 and/or TSC2 genes that result in the activation of mTOR pathway. Abnormally activated mTOR sends signals that encourage tumor cells to grow, metastasize, and invade new healthy tissues.

Fyarro (sirolimus protein-bound particles for injectable suspension) (albumin-bound)) is the first and only FDA approved therapy for the treatment of malignant PEComa. It is a targeted therapy that works to slow tumor growth by inhibiting mTOR.

Drugs used to treat Malignant Perivascular Epithelioid Cell Tumor