e16606 Background: To evaluate the efficacy and safety of neoadjuvant PD-1 inhibitor plus paclitaxel, pingyangmycin, and platinum-based chemotherapy ("4P" therapy) in high-risk non-muscle-invasive/muscle-invasive bladder cancer (N/MIBC), with a focus on bladder preservation and neoadjuvant optimization. Methods: This single-center, observational, retrospective study enrolled patients with cT1-4N0M0 N/MIBC from July 2022 to June 2024. Thirty-three patients received "4P" therapy followed by Transurethral Precise Resection of Bladder Tumor (TUPRBT). Radiological and pathological assessments were conducted every two cycles. The primary endpoint was the objective response rate (ORR); secondary endpoints included the disease control rate (DCR), complete response (CR), partial response (PR), and bladder preservation rate. Safety was evaluated according to CTCAE version 5.0 criteria. Results: Among the 33 patients (median age: 65 years; 87.8% male), ECOG performance status scores were 0 (78.8%) and 1 (21.2%). Clinical stages were T1 in 5 patients (15.2%) and T2-4 in 28 patients (84.8%). Patients were stratified by treatment cycles: 1 cycle (6 patients, 3 CR, 3 PR), 2 cycles (24 patients, 10 CR, 10 PR, 4 SD), and 3 cycles (3 patients, 3 PR), and the average treatment cycles were 1.91. The ORR for all patients was 87.8% (29/33), with a CR rate of 39.3% (13/33). DCR was 100% (33/33), with no cases of progressive disease. Among the different PD-1 inhibitor subgroups, the toripalimab-treated patients achieved an ORR of 92.0% (23/25) and a CR rate of 44.0% (11/25). Among the 26 patients who underwent TUPRBT, pCR was achieved in 9 (34.6%). At a median follow-up of 29.3 months, the median EFS was 29.3 months (95% CI: 22.8 – NE), with 1- and 2-year EFS rates of 84.5% (95% CI: 72.0–97.0%) and 65.5% (95% CI: 44.8–86.2%). The median OS was not reached, with 1- and 2-year OS rates of 93.7% (95% CI: 85.3–100%) and 81.2% (95% CI: 66.1–96.3%). The overall bladder preservation rate was 93.9% (31/33), as 2 patients ultimately underwent radical cystectomy. Disease recurrence occurred in 11 patients. Four deaths occurred (1 case of brain metastasis, 1 case of severe pneumonia, 2 cases of unspecified causes), of which only 1 was a bladder cancer-specific death. Treatment-related adverse events were primarily grade 1–2 and manageable, supporting the tolerability of the “4P” therapy. Conclusions: The "4P" regimen, with high efficacy and manageable toxicity, achieved a 93.9% bladder preservation rate in patients undergoing TUPRBT. By retaining bladder function and reducing surgical morbidity, it is an attractive choice for high-risk non-muscle-invasive and muscle-invasive bladder cancer patients' bladder preservation and neoadjuvant optimization.
Li et al. (Thu,) studied this question.