Pembrolizumab as neoadjuvant therapy before radical cystectomy in patients with muscle-invasive urothelial bladder carcinoma (PURE-01): An open-label, single-arm, phase II study

A Necchi, A Anichini, D Raggi, A Briganti, S Massa, R Lucianò, M Colecchia, P Giannatempo, R Mortarini, M Bianchi, Elena Farè, F Monopoli, R Colombo, A Gallina, A Salonia, A Messina, SM Ali, R Madison, JS Ross, JH ChungR Salvioni, L Mariani, F Montorsi

Research output: Contribution to journalArticle

Abstract

Purpose To determine the activity of pembrolizumab as neoadjuvant immunotherapy before radical cystectomy (RC) for muscle-invasive bladder carcinoma (MIBC) for which standard cisplatin-based chemotherapy is poorly used. Patients and Methods In the PURE-01 study, patients had a predominant urothelial carcinoma histology and clinical (c) T≤3bN0 stage tumor. They received three cycles of pembrolizumab 200 mg every 3 weeks before RC. The primary end point in the intention-to-treat population was pathologic complete response (pT0). Biomarker analyses included programmed death-ligand 1 (PD-L1) expression using the combined positive score (CPS; Dako 22C3 pharmDx assay), genomic sequencing (FoundationONE assay), and an immune gene expression assay. Results Fifty patients were enrolled from February 2017 to March 2018. Twenty-seven patients (54%) had cT3 tumor, 21 (42%) cT2 tumor, and two (4%) cT2-3N1 tumor. One patient (2%) experienced a grade 3 transaminase increase and discontinued pembrolizumab. All patients underwent RC; there were 21 patients with pT0 (42%; 95% CI, 28.2% to 56.8%). As a secondary end point, downstaging to pT,
Original languageEnglish
Pages (from-to)3353-3360
Number of pages8
JournalJournal of Clinical Oncology
Volume36
Issue number34
DOIs
Publication statusPublished - 2018

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    Necchi, A., Anichini, A., Raggi, D., Briganti, A., Massa, S., Lucianò, R., Colecchia, M., Giannatempo, P., Mortarini, R., Bianchi, M., Farè, E., Monopoli, F., Colombo, R., Gallina, A., Salonia, A., Messina, A., Ali, SM., Madison, R., Ross, JS., ... Montorsi, F. (2018). Pembrolizumab as neoadjuvant therapy before radical cystectomy in patients with muscle-invasive urothelial bladder carcinoma (PURE-01): An open-label, single-arm, phase II study. Journal of Clinical Oncology, 36(34), 3353-3360. https://doi.org/10.1200/JCO.18.01148