October 20, 2026Journal of Clinical OncologyCUPISCO (randomized phase 2), NCT03498521
Molecularly Guided Therapy Keeps Its Progression-Free Survival Edge Over Chemotherapy in Unfavorable Cancer of Unknown Primary, With No Statistically Significant Overall Survival Difference: Final Analysis of the Phase 2 CUPISCO Trial
CUPISCO randomly assigned 436 patients with newly diagnosed, unfavorable, nonsquamous cancer of unknown primary (CUP) who had disease control after three cycles of platinum-based induction chemotherapy, 3:1, to molecularly guided therapy (MGT; n = 326) or continued chemotherapy (n = 110). In this updated and final analysis (median follow-up 37.0 months), median progression-free survival (PFS) was 6.1 months with MGT versus 4.4 months with chemotherapy (HR 0.75; 95% CI, 0.59 to 0.95; P = .0177).