Perioperative chemotherapy in upper tract urothelial carcinoma: a comprehensive review

PURPOSE: To evaluate the role of neoadjuvant (NAC) and adjuvant chemotherapy (AC) in patients with upper tract urothelial carcinoma (UTUC) treated with radical nephroureterectomy (RNU).

METHODS: A comprehensive review of the current literature was performed searching for all studies investigating NAC and AC in UTUC in MEDLINE and https://clinicaltrials.gov , prior to April 2016. The following keywords were used: "ureteral neoplasms," "urothelium," "ureter," "upper tract urothelial," "chemotherapy," "adjuvant," "neoadjuvant" and relevant variants.

RESULTS: No randomized trials investigated the role of AC or NAC for UTUC. There was one prospective study with n = 36 patients investigating AC with carboplatin-paclitaxel. We included 14 retrospective studies (four in the NAC and ten in the AC setting), with a total of 694 patients receiving cisplatin-based or non-cisplatin-based AC after RNU and 1437 patients undergoing RNU alone. We found that the current literature, mainly based on retrospective studies, suggests significant overall and cancer-specific survival benefits for AC in UTUC. NAC appears promising, with favorable pathologic response rates up to 14%.

CONCLUSIONS: Evidence is scarce for both NAC and AC use in UTUC. This comprehensive review suggests promising response rates for NAC and a survival benefit for patients treated with AC. Prospective randomized trials are needed to establish the role of AC and NAC in UTUC.