The following summary is “Neoadjuvant Pembrolizumab and Radical Cystectomy in Patients with Muscle-Invasive Urothelial Bladder Cancer: 3-Year Median Follow-Up Update of PURE-01 Trial” published in the December 2022 issue of Oncology by Basilem et al.

Patients with muscle-invasive urothelial bladder cancer were the first to receive /neoadjuvant immune-checkpoint inhibitor (ICI) therapy prior to radical cystectomy (RC) in the PURE-01 study (NCT02736266) muscle-invasive urothelial bladder carcinoma(MIBC). After a median of 3 years of follow-up, researchers report the survival results here. A total of 155 people were included in the “intention to treat” (ITT) population analysis. Time to radiographic disease progression preventing RC, starting of neoadjuvant chemotherapy, recurrence after RC, or death was used to calculate event-free survival (EFS) from the start of pembrolizumab treatment. After radiotherapy and chemotherapy (RC), additional outcomes included progression-free survival (PFS) and overall survival (OS). 

The EFS was analyzed using multivariate Cox regression. Using Kaplan-Meier analysis, the EFS outcomes were compared by baseline programmed cell-death-ligand-1 (PD-L1) combined positive score (CPS) and molecular subtypes. Following a median (IQR) of 39 (30-47) months, the 36-month event-free survival (EFS) rate and overall survival (OS) rate in the ITT group were 74.4% [95% CI, 67.8-81.7] and 83.8% [95% CI, 77.8-90.2, respectively]. RC was performed on 143 individuals or 92.3% of the total. Patients who had ypT0N0, ypT1/a/isN0, ypT2-4N0, and ypTanyN1-3 responses had a 36-month RFS of 96.3% (95% CI, 91.6-100), 74.9% (95% CI, 60.2-93), and 58.3% (95% CI, 36.2-94.1), respectively, among the group of patients who did not receive further treatment (N=125).

Results for event-free survival (EFS) were significantly different amongst the 3 PD-L1 tertiles (lower tertile: 59.7%, medium tertile: 76.7%, and high tertile: 89.8%, P=0.0013). The occurrence rates were the lowest in the claudin-low and basal/squamous subtypes. PURE-01 data further show the long-term efficacy of neoadjuvant pembrolizumab before RC, with a median follow-up of 3 years. The expression of PD-L1 was the most reliable indicator of long-term survival after RC.

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