Abstract
Upper tract urothelial carcinoma (UTUC) is a very uncommon disease that occupies for <5% of all urothelial cancers. Radical nephroureterectomy (RNU) remains the standard-of-care for UTUC; however, when patients with locally advanced UTUC are treated with RNU only, the recurrence rate is high. Therefore, perioperative chemotherapy has been proposed given the high systemic recurrence rate. Moreover, there is growing evidence that neoadjuvant chemotherapy (NAC) plays an important role in the treatment of UTUC. Several studies and meta-analyses have reported the beneficial effect of NAC on survival outcomes and pathologic downstaging of patients with UTUC. However, the recommendation of NAC for UTUC is primarily based on level 1 evidence that demonstrated a beneficial effect on survival outcomes in patients with bladder cancer. The chemotherapy regimen for patients with UTUC is also based on that used for patients with bladder cancer. Nevertheless, the use of NAC for UTUC has some limitations, including the possibility of overtreatment. Therefore, selection criteria for NAC are needed, as are further trials to identify the most suitable patients and validate its use in daily clinical practice.