Neoadjuvant chemotherapy (NAC) may improve overall survival in patients with upper tract urothelial carcinoma (UTUC), according to investigators.

In a retrospective study of 10,315 patients with high-grade UTUC who underwent nephroureterectomy identified using 2004-2015 data from the National Cancer Data Base, 296 patients (2.9%) received preoperative NAC.

Use of NAC was significantly associated with superior  5-year survival (47% NAC vs 42% no NAC; P =.017), Douglas S. Scherr MD, of New York Presbyterian Hospital, Weill Cornell Medicine, New York, New York, and colleagues reported in Urology. When the team analyzed data by clinical stage, only patients with non-organ-confined tumors receiving NAC had significantly improved overall survival (P =.012). Of these patients, 27.1% had organ-confined disease at the time of surgery. In contrast, among patients without NAC, 1.4% had organ-confined disease at surgery.

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Significantly more patients who underwent NAC had pathologic downstaging: 10.8% NAC vs 1.4% no NAC; P <.001), according to Dr Scherr’s team.

On multivariate analysis, overall survival was significantly better for patients who received NAC (P =.044). Survival was significantly worse for older patients and those with higher pT stages, increasing Charlson-Deyo score (all P <.001), and treatment at a comprehensive community cancer program (P =.026).

“In a national contemporary cohort of patients who underwent [nephroureterectomy], administration of NAC was associated with improved survival across the entire cohort,” Dr Scherr and colleagues wrote. “However, the bulk of the survival advantage due to NAC administration appears to be a result of significant pathologic downstaging only in patients with significant disease burden at time of biopsy.”

Among the study’s limitations, tumor biology and chemotherapy regimens were both unknown. Selection bias also is inherent when patients are chosen for NAC. Lastly, the investigators could not assess cancer-specific survival using the database.


Khan AI, Taylor BL, Al Awamlh BAH, et al. Survival outcomes in neoadjuvant chemotherapy for high grade upper tract urothelial carcinoma: A nationally representative analysis. Urology. doi:10.1016/j.urology.2020.06.108