Multicenter phase II trial of ipilimumab and nivolumab in metastatic or unresectable perivascular epithelioid cell tumor (PEComa): a substudy of <i>D</i>ual <i>A</i>nti-CTLA-4 and Anti-PD-1 blockade in <i>R</i>are <i>T</i>umors (DART) SWOG S1609 (Cohort 38).

ABSTRACT:

Background

Malignant perivascular epithelioid cell tumors (PEComas) are ultra-rare, aggressive mesenchymal neoplasms associated with poor outcomes. Standard-of-care systemic therapy includes mammalian target of rapamycin (mTOR) inhibition and chemotherapy. Immune checkpoint inhibition has not been previously studied in patients with advanced PEComas in a prospective clinical trial.

Methods

This is an open-label phase 2 trial of ipilimumab (1 mg/kg intravenously every 6 weeks) plus nivolumab (240 mg intravenously every 2 weeks) in rare tumors; here we report the cohort of patients with advanced malignant PEComa recruited from >1,000 sites across the USA. Primary end point was objective response rate, with progression-free survival, overall survival, and toxicity as secondary

SUBMITTER: Patel SP 

PROVIDER: S-EPMC12542749 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature