Urachal cancer (UrC) is a rare disease which is mostly diagnosed late due to symptoms
caused by its local invasion to the urinary bladder. Given the lack of clinical trials
and guideline recommendations for systemic treatment, a molecularly informed precision
oncology approach is a viable option for UrC already in the early lines of systemic
treatment. While single case experiences may provide valuable reference for later
decision-making, well-documented clinical experience with off-label targeted treatments
is limited to a few patients. Here, we report a case of a 31-year-old female UrC patient
who underwent intensive therapy with three surgeries and five lines of systemic treatments,
including chemo-, checkpoint inhibitor and tyrosine kinase inhibitor therapies. In
addition, next-generation sequencing (NGS) analysis and an ex vivo drug-screening
analysis were performed on patient-derived tumor cells and the results were implemented
into the therapeutic decision-making. Finally, serum carcinoembryonic antigen (CEA)
levels proved to be helpful for therapy monitoring during the whole follow-up period.
