Bladder Cancer Coverage from Every Angle

Feasibility Study in Upper Tract Urothelial Cancer: Neoadjuvant Pembrolizumab Before Surgery

By: Kayci Reyer
Posted: Monday, August 16, 2021

Results from the PURE-02 study, presented in Urologic Oncology: Seminars and Original Investigations, suggest that neoadjuvant pembrolizumab monotherapy prior to radical nephroureterectomy does not appear to be an efficacious treatment for patients with high-risk localized upper tract urothelial carcinoma. Neoadjuvant pembrolizumab had previously been shown to trigger strong responses among patients with bladder urothelial carcinoma in the PURE-01 study; however, these results were unable to be replicated in PURE-02.

“Developing preoperative strategies with single-agent checkpoint inhibitors does not seem to be a reliable strategy in these rare and challenging-to-treat tumors,” concluded Francesco Montorsi, MD, FRCS (Hon), of Vita-Salute San Raffaele University, Milan, and colleagues.

Between August 2018 and November 2020, the study included 10 patients with upper tract urothelial carcinoma. Of those patients, nine (90%) completed neoadjuvant treatment, receiving 3 courses of 200 mg of intravenous pembrolizumab every 3 weeks before undergoing radical nephroureterectomy. A total of seven patients underwent radical nephroureterectomy, with one achieving a ypT1N0 response. That patient was believed to have had a cT1 tumor at the time of enrollment. Clinical complete response was achieved by one patient, who then refused radical nephroureterectomy. Disease progression occurred in two patients (20%), prompting the administration of chemotherapy prior to surgery.

Due to complications from severe myocarditis, myasthenia gravis, hepatitis, and myositis, one treatment-related death occurred. No association was identified between circulating tumor DNA and the likelihood of achieving a complete pathologic response.

Disclosure: For full disclosures of the study authors, visit

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