Introduction: To evaluate the overall survival and pathologic downstaging effect of neoadjuvant chemotherapy for upper tract urothelial cell carcinoma.
Materials and methods: The National Cancer Database (NCDB) was queried for patients with stage II-IV upper tract urothelial cell carcinoma undergoing definitive surgical resection (nephroureterectomy) from 2004-2015. Patients with metastatic disease were excluded. Cohorts were stratified by receipt of neoadjuvant chemotherapy (NAC). Kaplan-Meier analysis and Cox regression were used to evaluate overall survival. Logistic regression was used to predict the odds of pathologic downstaging to non-invasive disease (< pT2). Propensity score matched analysis was performed between groups.
Results: A total of 3634 patients were identified with non-metastatic stage II-IV disease undergoing surgical resection; 3364 received no chemotherapy and 270 received NAC. Patients undergoing NAC had a 10.9% rate of downstaging to non-invasive disease (OR 6.35, p < 0.001). Moreover, on Kaplan-Meier analysis, median survival was 27.3 months and 44.8 months for no chemotherapy versus NAC, respectively (log-rank, p = 0.001). Cox regression for death also revealed benefits for receiving NAC (HR 0.67, p < 0.001). Findings were confirmed on propensity score matching (532 matched patients). After matching, Cox regression for death noted improvement with neoadjuvant as compared to no chemotherapy (HR 0.61, p < 0.001).
Conclusion: Neoadjuvant chemotherapy increases likelihood of downstaging to non-invasive disease in patients with upper tract urothelial cell carcinoma. Chemotherapy also provides an overall survival benefit in patients undergoing nephroureterectomy.