Department of Cancer Medicine, University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Eur Urol. 2013 Aug;64(2):307-13. doi: 10.1016/j.eururo.2012.04.020. Epub 2012 Apr 17.
Small cell urothelial carcinoma (SCUC) is a rare, aggressive malignancy with a propensity for early microscopic metastases. Data suggest that neoadjuvant chemotherapy may lead to improved survival compared with initial surgery.
To determine the influence of neoadjuvant chemotherapy on survival of SCUC patients in a large single-institution cohort.
DESIGN, SETTING, AND PARTICIPANTS: Between 1985 and 2010, 172 patients were treated for SCUC at MD Anderson Cancer Center (MDACC). Clinical, pathologic, and surgical data were collected and analyzed.
Overall survival (OS) and disease-specific survival (DSS) were calculated using the Kaplan-Meier method. Multivariable Cox proportional hazards models were used to evaluate the effects of neoadjuvant chemotherapy on survival.
Of 125 patients with resectable disease (≤ cT4aN0M0), 95 were surgical candidates. Forty-eight received neoadjuvant chemotherapy, and 47 underwent initial surgery. Neoadjuvant treatment was associated with improved OS and DSS compared with initial cystectomy (median OS: 159.5 mo vs 18.3 mo, p<0.001; 5-yr DSS: 79% vs 20%, p<0.001). Neoadjuvant chemotherapy resulted in pathologic downstaging to ≤ pT1N0 in 62% of tumors compared with only 9% treated with initial surgery (odds ratio: 44.55; 95% confidence interval, 10.39-191). Eight patients with clinically node-positive disease had surgical consolidation with cystectomy and extended lymph node dissection after clinical complete response to chemotherapy. Median OS and DSS in this group of patients were 23.3 mo and 21.8 mo, respectively, with 5-yr OS and DSS of 38%.
Neoadjuvant chemotherapy is associated with a high rate of pathologic downstaging and correlates with significantly higher survival compared with historical expectations. Although limited by a small sample size and retrospective analysis, in the context of a rare disease, this experience suggests neoadjuvant chemotherapy as a standard approach in treating SCUC.
小细胞尿路上皮癌（SCUC）是一种罕见的侵袭性恶性肿瘤，具有早期显微镜下转移的倾向。数据表明，新辅助化疗可能比初始手术带来更好的生存获益。
在大型单机构队列中，确定新辅助化疗对 SCUC 患者生存的影响。
设计、地点和参与者：1985 年至 2010 年间，MD 安德森癌症中心（MDACC）共治疗了 172 例 SCUC 患者。收集并分析了临床、病理和手术数据。
使用 Kaplan-Meier 法计算总生存（OS）和疾病特异性生存（DSS）。多变量 Cox 比例风险模型用于评估新辅助化疗对生存的影响。
在 125 例可切除疾病（≤cT4aN0M0）患者中，95 例为手术候选者。48 例接受了新辅助化疗，47 例接受了初始手术。与初始膀胱切除术相比，新辅助治疗改善了 OS 和 DSS（中位 OS：159.5 个月 vs 18.3 个月，p<0.001；5 年 DSS：79% vs 20%，p<0.001）。新辅助化疗使 62%的肿瘤降期至≤pT1N0，而初始手术仅为 9%（优势比：44.55；95%置信区间，10.39-191）。8 例临床淋巴结阳性疾病患者在接受化疗后出现临床完全缓解，随后进行了膀胱切除术和扩大淋巴结清扫术。该组患者的中位 OS 和 DSS 分别为 23.3 个月和 21.8 个月，5 年 OS 和 DSS 分别为 38%。
新辅助化疗与较高的病理降期率相关，并与明显高于历史预期的生存相关。尽管受到样本量小和回顾性分析的限制，但在罕见疾病的背景下，这一经验表明新辅助化疗是治疗 SCUC 的标准方法。
