Pleomorphic rhabdomyosarcoma, outcomes of patients with advanced disease treated with systemic agents: Retrospective study from the global pushing ultra-rare sarcomas towards hope (PUSH) consortium

This series of advanced P-RMS treated with systemic agents, the largest available to date, showed meaningful activity of anthracycline- and gemcitabine-based regimens, and anecdotal responses to pazopanib and PD-1 inhibitors. Further prospective validation is planned.