Background: Plasmacytoid urothelial carcinoma (PUC) is a rare histologic subtype of urothelial carcinoma of the bladder (BC). Our objective was to characterize treatment patterns and outcomes of PUC in the NCDB and our recent institutional experience. Methods: The NCDB was queried for localized PUC cases between 2004 and 2020. Patients with PUC from a single institution (Yale School of Medicine) were also incorporated from 2021 onwards to not double-count patients. The primary outcomes were overall survival and treatment trends. Results: A total of 146 patients were included, 123 from NCDB and 23 from Yale. The median overall survival (mOS) was 28 [IQR 7.5, 50.3] months, 23 [IQR 8.4, 46.3] months for the NCDB patients, and 36 [IQR 4.3, 68.1] for the Yale patients. The mOS for patients receiving neoadjuvant chemotherapy (NAC) was 60.0 [28.0, 91.9] vs. 14.8 months [0, 34.3] for patients without NAC,p= 0.038, though the benefit was not preserved in a Cox proportional hazard analysis incorporating the clinical stage, receipt of NAC, and age. The peritoneum was the most common site of metastasis (78.3%), followed by the liver and bones. Conclusion: Our findings underscore the formidable challenge posed by PUC, emphasizing its limited response to current therapies. Despite higher pT0 rates with NAC, the OS benefit remains inconclusive, highlighting the need for more effective treatments.
背景：浆细胞样尿路上皮癌（PUC）是膀胱尿路上皮癌（BC）中一种罕见的组织学亚型。本研究旨在通过美国国家癌症数据库（NCDB）及本机构近期临床经验，分析PUC的治疗模式与临床结局。方法：检索NCDB中2004年至2020年局部PUC病例数据，并纳入2021年起耶鲁大学医学院单中心PUC病例以避免重复计数。主要研究终点为总生存期及治疗趋势。结果：共纳入146例患者，其中NCDB数据库123例，耶鲁中心23例。总体中位总生存期（mOS）为28个月[四分位距7.5, 50.3]，NCDB患者为23个月[8.4, 46.3]，耶鲁患者为36个月[4.3, 68.1]。接受新辅助化疗（NAC）患者的mOS为60.0个月[28.0, 91.9]，未接受NAC者为14.8个月[0, 34.3]（p=0.038），但在纳入临床分期、NAC使用情况及年龄的Cox比例风险模型中，该生存获益未保持统计学显著性。腹膜是最常见转移部位（78.3%），其次为肝脏与骨骼。结论：本研究结果凸显PUC治疗的严峻挑战，提示其对现有治疗方案反应有限。尽管NAC可获得更高的病理完全缓解率，但其总生存获益尚未明确，亟需探索更有效的治疗策略。
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