KEY TAKEAWAYS
- The PURE-01 phase 2 study compared pathologic response and survival outcomes in cisplatin-ineligible patients receiving NAP vs. IRC.
- OS was analyzed through the Kaplan-Meier method and Log-Rank functions.
- The variable contribution to survival was measured using the Cox-proportional hazards model.
- Cisplatin-ineligible patients receiving NAP for MIBC showed significantly better downstaging and survival over those who underwent IRC.
In this study, patients (pts) ineligible for cisplatin and with cT2-4N0M0 muscle-invasive bladder cancer (MIBC) were identified from the PURE-01 trial and evaluated against cisplatin-ineligible MIBC patients who had received immediate radical cystectomy (IRC). Overall survival (OS) was analyzed through the Kaplan-Meier method and Log-Rank functions. The study utilized the Cox-proportional hazards model to determine the variable contribution to survival. Propensity score matching was also conducted for IRC and neoadjuvant pembrolizumab (NAP) pts based on assessments like pre-cystectomy ECOG status, GFR, age, sex, and clinical T stage. An additional exploratory analysis was done to match pts by pathologic T stage. All statistical analysis was performed using R.
In the PURE-01 study, 39 patients who were not eligible for cisplatin treatment were treated with NAP, and compared to 313 cisplatin-ineligible patients who received IRC. The results indicated that those who received NAP had a significantly longer overall survival (OS), with a median survival of not reached (NR) compared to 19 months in the IRC group. This difference was statistically significant (p<0.01). After conducting a propensity match, 39 patients were identified from the IRC cohort with similar clinicopathologic data. Those who received NAP had a higher complete response rate on final pathology than those who did not (pT0: 33% vs. 13%, p=0.03). Additionally, the NAP group had a longer median overall survival (OS) compared to the IRC group (NR vs. 21.0 mo, p<0.01), with significant differences at 12 mo (89% vs. 57%), 24mo (64% vs. 28%), and 36 mo post-surgery (33% vs. 13% ) (p<0.01). Cox Proportional Hazards Modelling showed that patients who underwent IRC had worse OS with a hazard ratio of 2.0 (95% CI 1.1 – 3.89).
Cisplatin-ineligible pts receiving NAP for MIBC demonstrated a higher rate of downstaging and survival advantage over those who underwent IRC. Ongoing prospective randomized trials are underway to validate these findings and provide therapeutic options to patients who cannot tolerate cisplatin.
Source: https://www.auajournals.org/doi/10.1097/JU.0000000000003309.09
Clinical Trial: https://classic.clinicaltrials.gov/ct2/show/NCT02736266
Rose, Kyle; Bandini, Marco; Huelster, Heather; Basile, Giuseppe; Naidu, Shreyas; Spiess, Philippe; Necchi, Andrea; Li, Roger MP56-09 NEOADJUVANT PEMBROLIZUMAB SHOWS PROMISE AS EFFECTIVE SYSTEMIC THERAPY PRIOR TO RADICAL CYSTECTOMY FOR CISPLATIN-INELIGIBLE MUSCLE INVASIVE BLADDER CANCER, Journal of Urology: April 2023 – Volume 209 – Issue Supplement 4
doi: 10.1097/JU.0000000000003309.09
