Adjuvant chemotherapy in upper tract urothelial carcinoma (the POUT trial): a phase 3, open-label, randomised controlled trial

In this study (n=261), adjuvant chemotherapy (platinum plus gemcitabine) was associated with improved disease-free survival (3-year event-free estimates of 71% (95% CI 61–78) vs 46% (36–56) for surveillance, HR 0.45; 95% CI 0.30-0.68; p=0.0001).

SPS commentary:

A related comment notes that this is the first dedicated randomised Phase III trial of perioperative chemotherapy in upper tract urothelial (transitional cell) carcinoma (UTUC), which is a rare disease (occurring in roughly two people per 100,000 population).

Due to the rarity of the condition, eligibility criteria were made as pragmatic as possible. Disease-free survival was selected as the primary endpoint, as a suitably powered trial with overall survival as the primary endpoint was not considered feasible. It is noted however that validation of disease-free survival as a surrogate for overall survival benefit is not yet formally established, and uro-oncologists need to decide whether disease-free survival benefit represents an appropriate bar for practice change. Mature overall survival data (secondary endpoint) are awaited.


The Lancet

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