This study aimed to evaluate the outcomes and identify the predictive factors of a bladder-preservation approach incorporating maximal transurethral resection of bladder tumor (TURBT) coupled with either pembrolizumab or chemotherapy for patients diagnosed with muscle-invasive bladder cancer (MIBC) who opted against definitive local therapy. We conducted a retrospective analysis on 53 MIBC (cT2-T3N0M0) patients who initially planned for neoadjuvant pembrolizumab or chemotherapy after maximal TURBT but later declined radical cystectomy and radiotherapy. Post-therapy clinical restaging and conservative bladder-preservation measures were employed. Clinical complete remission was defined as negative findings on cystoscopy with biopsy confirming the absence of malignancy if performed, negative urine cytology, and unremarkable cross-sectional imaging (either CT scan or MRI) following neoadjuvant therapy. Twenty-three patients received pembrolizumab, while thirty received chemotherapy. Our findings revealed that twenty-three (43.4%) patients achieved clinical complete response after neoadjuvant therapy. The complete remission rate was marginally higher in pembrolizumab group in comparison to chemotherapy group (52.1% vs. 36.7%,p= 0.26). After a median follow-up of 37.6 months, patients in the pembrolizumab group demonstrated a longer PFS (median, not reached vs. 20.2 months,p= 0.078) and OS (median, not reached vs. 26.8 months,p= 0.027) relative to those in chemotherapy group. Those achieving clinical complete remission post-neoadjuvant therapy also exhibited prolonged PFS (median, not reached vs. 10.2 months,p< 0.001) and OS (median, not reached vs. 24.4 months,p= 0.004). In the multivariate analysis, clinical complete remission subsequent to neoadjuvant therapy was independently associated with superior PFS and OS. In conclusion, bladder preservation emerges as a viable therapeutic strategy for a carefully selected cohort of MIBC patients without definitive local therapy, especially those achieving clinical complete remission following neoadjuvant treatment. For patients unfit for chemotherapy, pembrolizumab offers a promising alternative treatment option.
本研究旨在评估针对拒绝接受根治性局部治疗的肌层浸润性膀胱癌（MIBC）患者，采用经尿道膀胱肿瘤最大程度切除术（TURBT）联合帕博利珠单抗或化疗的保膀胱治疗方案的疗效，并分析其预测因素。我们对53例初始计划在最大程度TURBT后接受新辅助帕博利珠单抗或化疗，但后续拒绝根治性膀胱切除术及放疗的MIBC（cT2-T3N0M0）患者进行了回顾性分析。治疗后采用临床再分期及保守性保膀胱措施。临床完全缓解定义为新辅助治疗后膀胱镜检查阴性（若行活检则病理证实无恶性病变）、尿细胞学检查阴性及横断面影像学检查（CT或MRI）未见异常。其中23例患者接受帕博利珠单抗治疗，30例接受化疗。结果显示，23例（43.4%）患者在新辅助治疗后达到临床完全缓解。帕博利珠单抗组的完全缓解率略高于化疗组（52.1% vs. 36.7%，p=0.26）。中位随访37.6个月后，与化疗组相比，帕博利珠单抗组患者表现出更长的无进展生存期（中位数未达到 vs. 20.2个月，p=0.078）和总生存期（中位数未达到 vs. 26.8个月，p=0.027）。新辅助治疗后获得临床完全缓解的患者亦显示出更长的无进展生存期（中位数未达到 vs. 10.2个月，p<0.001）和总生存期（中位数未达到 vs. 24.4个月，p=0.004）。多变量分析表明，新辅助治疗后的临床完全缓解与更优的无进展生存期和总生存期独立相关。综上所述，对于经过严格筛选、未接受根治性局部治疗的MIBC患者群体，尤其是新辅助治疗后达到临床完全缓解者，保膀胱治疗是一种可行的治疗策略。对于不适合化疗的患者，帕博利珠单抗提供了一种有前景的替代治疗方案。
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