The role of neoadjuvant systemic therapy for high grade upper tract urothelial carcinoma: Results from the upper tract collaborative network (UCAN) Journal Article


Authors: Carpinito, G. P.; Gerald, T.; Hensley, P. J.; Martin, A. J.; Pallauf, M.; Pham, J.; Li, R.; Potretzke, A. M.; Spiess, P. E.; Singla, N.; Raman, J. D.; Coleman, J.; Matin, S. F.; Margulis, V.
Article Title: The role of neoadjuvant systemic therapy for high grade upper tract urothelial carcinoma: Results from the upper tract collaborative network (UCAN)
Abstract: Introduction: Utilization of neoadjuvant systemic therapy (NAT) prior to radical nephroureterectomy (RNU) for upper tract urothelial carcinoma (UTUC) is inconsistent, and optimal patient selection for NAT is unclear. The purpose of this study was to evaluate the clinical benefit of NAT in high grade UTUC undergoing RNU. Materials and Methods: The UTUC Collaborative Network (UCAN) identified patients who underwent RNU for high grade UTUC between 2000 and 2022. NAT was examined as a primary exposure. NAT was defined as any systemic therapy prior to RNU. The outcomes of interest were extra-urothelial recurrence free survival (euRFS), cancer-specific survival (CSS), and overall survival (OS). Results: Among 461 patients meeting criteria, 51.2% received NAT. At a median follow-up of 2.9 years, 24.1% experienced extra-urothelial recurrence at a median of 2.4 (1.0-5.2) years. On multivariable Cox proportional hazards models, NAT was associated with improved CSS (HR 0.58; 95% CI 0.36-0.94). In clinically node negative patients receiving NAT, Kaplan-Meier analysis showed improved euRFS (P = 0.01), cancer-specific survival (P = 0.002), and overall survival (P = 0.002). A statistically significant benefit was not observed for clinically node positive patients receiving NAT in euRFS (P = 0.667), CSS (P = 0.200), or OS (P = 0.313). Conclusions: NAT was associated with improved survival outcomes in patients with clinically node negative disease. These benefits were not consistently observed in those with clinically node positive disease, although there was trend toward improved outcomes on multivariable Cox models. Further prospective investigations regarding risk stratification and multimodal management are needed in patients with high grade UTUC. © 2024 Elsevier Inc.
Keywords: survival; controlled study; aged; middle aged; retrospective studies; major clinical study; overall survival; mortality; cancer recurrence; cisplatin; doxorubicin; systemic therapy; gemcitabine; paclitaxel; neoadjuvant therapy; methotrexate; follow up; cancer grading; cohort analysis; recurrence; pathology; retrospective study; kidney neoplasms; ifosfamide; vinblastine; kidney tumor; cancer specific survival; multicenter study; nephroureterectomy; surgery; upper tract urothelial carcinoma; ureteral neoplasms; kaplan meier method; drug therapy; carcinoma, transitional cell; transitional cell carcinoma; ureter tumor; recurrence free survival; neoadjuvant treatment; clinical outcome; procedures; adjuvant treatment; neoplasm grading; humans; human; male; female; article
Journal Title: Urologic Oncology: Seminars and Original Investigations
Volume: 43
Issue: 6
ISSN: 1078-1439
Publisher: Elsevier Inc.  
Date Published: 2025-06-01
Start Page: 390.e1
End Page: 390.e11
Language: English
DOI: 10.1016/j.urolonc.2024.11.025
PUBMED: 39721824
PROVIDER: scopus
DOI/URL:
Notes: Source: Scopus
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  1. Jonathan Coleman
    341 Coleman