Trends in the use of neoadjuvant chemotherapy and oncological outcomes for high‐risk upper tract urothelial carcinoma: a multicentre retrospective study

Objective To evaluate temporal trends in neoadjuvant chemotherapy (NAC) utilisation and outcomes in patients with locally advanced upper tract urothelial carcinoma (UTUC). Patients and Methods We included 289 patients from seven hospitals who underwent radical nephroureterectomy (RNU) for locally advanced UTUC (≥cT3 or cN+) between 2000 and 2020. These patients received RNU alone or two to four courses of NAC with either a cisplatin‐ or carboplatin‐based regimen. We evaluated the temporal changes in NAC use and compared the visceral recurrence‐free, cancer‐specific, and overall survival rates. The effect of NAC on oncological outcomes was examined using multivariate Cox regression analysis with inverse probability of treatment weighting (IPTW) models. Results Of 289 patients, 144 underwent NAC followed by RNU (NAC group) and 145 underwent RNU alone (Control [Ctrl] group). NAC use increased significantly from 19% (2006–2010), 58% (2011–2015), to 79% (2016–2020). Pathological downstaging was significantly higher in the NAC group than in the Ctrl group. The IPTW‐adjusted multivariable analyses showed that NAC significantly improved the oncological outcomes in the NAC group compared with the Ctrl group. Moreover, carboplatin‐based NAC significantly improved the oncological outcomes in the NAC group compared with the Ctrl group among patients with chronic kidney disease Stage ≥3. There were no significant differences in oncological outcomes between the cisplatin‐ and carboplatin‐based regimens. Conclusions The use of NAC for high‐risk UTUC increased significantly after 2010. Platinum‐based short‐term NAC followed by immediate RNU may not impede and potentially improves oncological outcomes.