The PURE-01 trial evaluated three cycles of pembrolizumab 200 mg followed by radical cystectomy (RC) in patients with cT2-3b N0 M0 muscle-invasive bladder cancer (MIBC) ineligible for or refusing cisplatin.
A total of 155 patients were treated. We report survival outcomes after median follow-up of >60 mo and associations with pathological response rates and biomarker levels. In the intention-to-treat population, the 5-yr event-free survival rate was 68% and the 5-yr overall survival (OS) rate was 77%. In the RC cohort, the 5-yr recurrence-free survival (RFS) rate was 77%. Pathological response categories were significantly associated with survival, with a 5-yr OS rate of ∼90% in the group achieving a complete or major response (p < 0.001). The 5-yr cumulative risk of recurrence was 19%, with three of 31 relapses occurring beyond 5 yr after RC. Eight patients refused RC and underwent transurethral resection; seven remain alive and disease-free. Transcriptome-wide profiling of pretreatment tissue was available for 102 patients. Stratification by genomic subtyping classifier showed that the claudin-low subtype (n = 14) had the highest 5-yr RFS rate, with a 5-yr OS rate of 93%. At long-term follow-up, PURE-01 demonstrates sustained survival outcomes and confirms the prognostic relevance of pathological response and molecular subtypes. These results support further investigation of single-agent immunotherapy as a potential de-escalation strategy in selected patients.
European urology. 2026 Feb 02 [Epub ahead of print]
Valentina Tateo, Giuseppe Basile, Patrizia Giannatempo, Joep J de Jong, James A Proudfoot, Brigida A Maiorano, Antonio Cigliola, Chiara Mercinelli, Elai Davicioni, Marco Moschini, Giovanni Tremolada, Simone Rota, Giorgio Brembilla, Maurizio Colecchia, Francesco De Cobelli, Francesco Montorsi, Andrea Necchi
Department of Medical Oncology, Comprehensive Cancer Center, IRCCS San Raffaele Hospital, Milan, Italy. Electronic address: ., Department of Urology, IRCCS Ospedale San Raffaele, Milan, Italy., Department of Medical Oncology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy., Department of Urology, Erasmus University Medical Center, Rotterdam, Netherlands., Veracyte, Inc., South San Francisco, CA, USA., Department of Medical Oncology, Comprehensive Cancer Center, IRCCS San Raffaele Hospital, Milan, Italy., Department of Medical Oncology, Comprehensive Cancer Center, IRCCS San Raffaele Hospital, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy., Department of Urology, IRCCS Ospedale San Raffaele, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy., Vita-Salute San Raffaele University, Milan, Italy; Department of Radiology, IRCCS Ospedale San Raffaele, Milan, Italy., Vita-Salute San Raffaele University, Milan, Italy; Department of Pathology, IRCCS Ospedale San Raffaele, Milan, Italy.
PubMed http://www.ncbi.nlm.nih.gov/pubmed/41633898