AUA 2022: Neoadjuvant Pembrolizumab May Result in Long-Term Outcome Improvement in Biomarker-Unselected Patients With Muscle-Invasive Bladder Cancer: Updated results from PURE-01

(  The 2022 Annual Meeting of the American Urological Association was host to a moderated poster session for non-invasive bladder cancer. Dr. Luigi Nocera provided updated outcomes results of PURE-01, an open-label, single-arm, phase 2 study, where cT2-4aN0M0 MIBC patients received three courses of 200 mg pembrolizumab preceding radical cystectomy. Pathologic response rates have been reported previously in this study with pT0 and <=pT1 rates of 37% and 55%, respectively. The aim of this report is to present updated results on recurrence-free survival (RFS) and overall survival (OS) at longer follow-up.


136 patients were treated between February 2017 and June 2020. RFS was defined as a composite of:

  • Radiologic progression
  • Local or distant recurrence
  • Death from any cause

 Survival analysis utilizing Kaplan-Meier analyses and Cox regression models was performed.

Of the 136 patients, 120 (88%) were male and median age was 68.0 years (IQR 62.0-74.0). PD-L1 CPS=10% on the TURB specimen pre-therapy was seen in 76 patients (56%).


Radical cystectomy pathologic evaluation demonstrated:

  • ypT0N0: 57 (42%)
  • ypTa/1/isN0: 25 (18%)
  • ypT2-4N0: 34 (25%)
  • ypTanyN+: 20 (15%)

 Nine patients received adjuvant chemotherapy: 3 ypT2-4N0 and 6 ypTanyN+ patients. After a median follow-up of 27 months (IQR 21-38), recurrence was observed in 22 patients (16%), while 14 patients (10%) died.


36-month OS was:

  • ypT0N0: 95%
  • ypTa/1/isN0: 86%
  • ypT2-4N0: 93%
  • ypTanyN+: 56%


36-month RFS:

  • ypT0N0: 94%
  • ypTa/1/isN0: 96%
  • ypT2-4N0: 67%
  • ypTanyN+: 44%



In ITT population, 36-month RFS and OS for CPS=10% vs CPS>10 cohorts were respectively 88% vs 72% and 91% vs 79%. On multivariable Cox regression analyses, predictors of:


Overall mortality:

  • Age: HR 1.1, p=0.02
  • ypTanyN+ stage: HR 9.0, p=0.02



  • ypT2-4N0: HR 8.6, p=0.007
  • ypTanyN+: HR 15.9, p=0.001




 The authors concluded that neoadjuvant pembrolizumab followed by radical cystectomy is an effective treatment for patients with MIBC, unselected for biomarkers and for cisplatin eligibility. This data should be tested towards the data emerging in adjuvant setting and in light of the ongoing randomized perioperative phase 3 trials.

Presented by: Luigi Nocera, MD, Resident Physician, Department of Urology, San Raffaele Scientific Institute, Milan, Italy

Written by: Rashid Sayyid, MD, MSc – Urology Chief Resident, Augusta University/Medical College of Georgia, @rksayyid on Twitter during the 2022 American Urological Association (AUA) Annual Meeting, New Orleans, LA, Fri, May 13 – Mon, May 16, 2022.