Introduction: Cancer of unknown primary (CUP) is a metastatic cancer that evades a primary site diagnosis and is the 6th most common cause of cancer-related death in Australia. CUP patients have difficulty in accessing precision treatments given tissue of origin (TOO) is often a prerequisite. Whole genome and transcriptome sequencing (WGTS) can help resolve a TOO and identify precision treatments in CUP, but requires validation in a real-world setting.
Study design: Molecular testing was applied to CUP patients from 12 Australian sites using gene-panel (Illumina TSO500) and/or WGTS. Patient eligibility included a standardised diagnostic work-up (ESMO guidelines). Patients were excluded if they had a poor ECOG (>2). Curated molecular reports were delivered to the treating physician and pathologists via a molecular tumour board. Diagnostic impact of testing was assessed by surveying pathologists and treating clinicians.
Results: We recruited 203 patients overs 18 months to the SUPER-NEXT study. For molecular testing, 90% of cases involved formalin-fixed paraffin embedded (FFPE) tissues; predominantly core tissue biopsies (81.5%). Fifty-three percent (107/203) were suitable for both WGTS and TSO500 cancer panel. Twenty-six percent (53/203) of cases were suitable for TSO500 only and 18% (36/203) received no test. Additional reportable variants were found by WGTS in 85.7% of cases receiving both tests. An algorithmic TOO classifier (CUPPA) was applied to WGTS data and predicted TOO with high-likelihood in 52% of cases. Based on pathologist surveys, a WGTS+CUPPA result impacted the diagnostic opinion for 80% (86/107) of cases and assisted single site TOO diagnosis in 64% (68/107) that received WGTS+CUPPA compared with only 38% (17/45) of cases receiving TSO500 only.
Conclusion: Clinical WGTS was possible in more than half of patients with CUP despite use of FFPE samples. WGTS was superior to TSO500 panel in resolving a cancer diagnosis. This data supports the utility WGTS as part of a histopathology work up for CUP patients.