Platinum-based Neoadjuvant Chemotherapy Improves Oncological Outcomes in Patients with Locally Advanced Upper Tract Urothelial Carcinoma.

BACKGROUND: Neoadjuvant chemotherapy (NAC) use for patients with locally advanced upper tract urothelial carcinoma (UTUC) is debatable.

OBJECTIVE: To investigate the efficacy and safety of platinum-based NAC for locally advanced UTUC.

DESIGN, SETTINGS, AND PARTICIPANTS: Of 233 consecutive patients who underwent radical nephroureterectomy, 55 patients received NAC (NAC group) and 138 patients did not (Ctrl group).

OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS: The two arms (Ctrl vs NAC) were matched using propensity scores to minimize selection bias. We retrospectively evaluated tumor response, post-therapy pathological downstaging, lymphovascular invasion, Ki67 status, and prognosis between pair-matched patients. Multivariate Cox regression analysis was performed for independent factors for prognosis.

RESULTS AND LIMITATIONS: We selected 51 pair-matched patients in each group. The regimens in the NAC group included gemcitabine and carboplatin, and gemcitabine and cisplatin. The median response rate in the NAC group was 28%. NAC-related adverse events were tolerable. Pathological downstaging of the primary tumor was significantly higher in the NAC group than in the Ctrl group. The MIB1 index (immunostaining for Ki67) was significantly higher in the NAC group. NAC for locally advanced UTUC significantly prolonged progression-free, cancer-specific, and overall survival. Multivariate Cox regression analysis using an inverse probability of treatment weighting method showed that NAC was selected as an independent predictor for prolonged cancer-specific survival. Limitations are the retrospective design and the small sample size.

CONCLUSIONS: Platinum-based NAC for advanced UTUC potentially improves oncological outcomes. Further prospective studies are needed.

PATIENT SUMMARY: Platinum-based neoadjuvant chemotherapy for locally advanced upper tract urothelial carcinoma was safe and potentially improves oncological outcomes. A carboplatin-based regimen may be used as an alternative in patients with impaired renal function.