Single-center randomized trial of site-specific therapy guided by 90-gene expression assay versus empirical chemotherapy for cancer of unknown primary

Empirical chemotherapy is the standard of care in patients with unfavorable cancer of unknown primary (CUP). Gene-expression profiling assays have been developed to identify the tissue of origin in patients with CUP; however, their clinical benefit has not yet been demonstrated. The randomized Fudan CUP-001 trial, at Fudan University (Shanghai, China) compared site-specific therapy guided ty a 90-gene expression assay versus empirical chemotherapy in patients with CUP. The study included patients with previously untreated CUP and an ECOG performance status 0-2, who were randomized 1:1 to site-specific therapy or empirical chemotherapy. The median progression-free survival (primary endpoint) was 9.6 months in the group with site-specific therapy versus 6.6 months in the group with empirical chemotherapy (HR 0.68; p=0.017). Grade 3 or 4 treatment-related adverse events were seen in 56% of patients in the site-specific therapy group and 61% of patients in the empirical chemotherapy group. There were no grade 5 TRAEs. The authos conclude that site-specific therapy guided by the 90-gene expression assay was associated with better PFS when compared with empirical chemotherapy among patients with CUP.