A new treatment regimen using enfortumab vedotin, either as monotherapy or in combination with pembrolizumab, has demonstrated promising efficacy for patients with upper tract urothelial cancer who are typically ineligible for standard cisplatin chemotherapy.
The findings, presented at the ASCO GU symposium, emerged from a retrospective analysis focusing on patients with locally advanced and metastatic primary upper tract urothelial cancer. The study addresses a critical gap in treatment options for this patient population, who are often excluded from clinical trials due to comorbidities or surgery-related renal function issues.
Treatment Outcomes and Patient Demographics
In the analysis of 22 patients, researchers observed an objective response rate of 36.4%. More impressively, the disease control rate - defined as the sum of complete response, partial response, and stable disease - reached 72.3%. The study population predominantly consisted of patients with visceral metastases at baseline (81.8%), representing a particularly challenging-to-treat group.
Clinical Significance and Expert Perspective
Dr. Evangelia Vlachou, a postdoctoral research fellow at Johns Hopkins Medicine, emphasized the importance of these findings. "These patients are often capped or excluded from urothelial cancer trials, particularly in the perioperative setting," she explained. "We felt that an important question to ask is whether enfortumab and enfortumab with pembrolizumab, which has revolutionized the management of the advanced setting, can also be used in the perioperative setting."
Current Treatment Landscape
While cisplatin chemotherapy remains the standard first-line treatment for invasive urothelial cancer in the perioperative setting, many upper tract urothelial cancer patients are ineligible for this approach. The new regimen potentially offers an alternative pathway for these previously limited patients.
Future Research Directions
Researchers acknowledge the need for larger, prospective studies to validate these findings. A forthcoming analysis from the phase 3 global trial EV-302, led by Dr. Thomas Powles, will provide additional insights into the combination of enfortumab vedotin and pembrolizumab in previously untreated locally advanced or metastatic urothelial carcinoma, with data from 886 randomized patients.
