Retrospective study of anti-PD-1 therapy with or without chemotherapy for refractory or relapsed gestational trophoblastic neoplasia

A retrospective study at three centers in China evaluated anti-PD-1 therapy with or without chemotherapy for high-risk chemorefractory or relapsed gestational trophoblastic neoplasia. The combined treatment dramatically increased the objective response rate from 62.9% with anti-PD-1 therapy alone to 96.8% with anti-PD-1 therapy plus chemotherapy (p<0.001). The progression-free survival was significantly longer with anti-PD-1 therapy plus chemotherapy than with anti-PD-1 therapy alone (HR 0.06; p<0.001). Treatment-related adverse events were observed in 80.6% of patients receiving anti-PD-1 therapy plus chemotherapy and 77.1% of patients receiving anti-PD-1 therapy alone (p=0.729). Among patients not responding to anti-PD-1 therapy alone, 84.6% responded to salvage chemotherapy.