Multicenter phase 2 study of neoadjuvant gemcitabine plus cisplatin for high-grade upper tract urothelial carcinoma

High-risk upper tract urothelial carcinoma (UTUC) has a poor prognosis. Neoadjuvant chemotherapy was associated with survival benefit among patients with invasive urothelial carcinoma of the bladder, but its role in UTUC is unclear. A multicenter phase 2 trial in the USA evaluated neoadjuvant gemcitabine plus split-dose cisplatin for high risk UTUC, before extirpative surgery. The neoadjuvant therapy was well-tolerated, and resulted in 5-year progression free survival rate of 72% and 5-year overall survival rate of 79%. The authors conclude that these data support the use of neoadjuvant chemotherapy as a standard of care for high-risk UTUC.