Perivascular epithelioid cell tumors (PEComa) is a rare subset of soft tissue sarcomas. PEComas appear to arise most commonly at gastrointestinal, uterine, abdominal, and pelvic sites. Due to the extremely rare nature of malignant PEComa, there is very limited information on its incidence. It is estimated that there are roughly 100 new patients per year in the US with malignant PEComa, however, since the disease is likely to be under-diagnosed or mis-diagnosed, the numbers could be substantially higher.
Most PEComas are benign, however there is a subset of aggressive PEComas (or advanced malignant PEComas), that may be either metastatic or locally advanced. Within these subsets surgery is not an option and there are currently no approved therapies
Based on retrospective analyses, data suggests that a mix of patients have been treated with chemotherapy, radiotherapy, and mTOR inhibitors. Therefore, a significant unmet medical need exists for effective therapies to treat this aggressive disease.