Importance  Small cell carcinoma of the urinary bladder is a rare and aggressive neuroendocrine tumor of the urinary bladder. Although research has been performed since the first case was reported in 1981, most of our understanding of the disease treatments has been extrapolated from small cell carcinoma of the lung. However, current data on patient survival have been stagnant.

Observations  With the advent of advanced molecular diagnostic methods, a new potential for understanding the origin and treatment of small cell carcinoma of the urinary bladder has become evident. Similar to published studies of small cell carcinoma of the lung, collaborative efforts and accessible genome-phenome databases have partially elucidated the genomic landscape. With these efforts, additional potential areas of treatment, such as targeting driver mutations, immunotherapy, stem cell modulation, and novel drug candidates, have been identified. Moreover, identification of novel biomarkers and improved radiological methods may complement our understanding of the course of disease relative to diagnosis, staging, prognosis, and response to treatment. Such early discoveries have been facilitated with novel animal-based models.

Conclusions and Relevance  The direction of investigation of small cell carcinoma of the urinary bladder using novel methods to understand the genomic landscape for new treatments remains preliminary. However, such studies are maturing and may foster clinical research, with opportunities to enhance the survival and health-related quality of life of individuals with this disease.

重要性  膀胱小细胞癌是一种罕见的侵袭性膀胱神经内分泌肿瘤。尽管自 1981 年报告第一例病例以来已经进行了研究，但我们对疾病治疗的大部分理解都是从肺小细胞癌中推断出来的。然而，目前关于患者生存的数据一直停滞不前。

观察  随着先进分子诊断方法的出现，了解膀胱小细胞癌的起源和治疗的新潜力已变得明显。与已发表的肺小细胞癌研究类似，协作努力和可访问的基因组表型数据库已经部分阐明了基因组景观。通过这些努力，已经确定了其他潜在的治疗领域，例如靶向驱动突变、免疫疗法、干细胞调节和新的候选药物。此外，新生物标志物的鉴定和改进的放射学方法可以补充我们对与诊断、分期、预后和治疗反应相关的疾病过程的理解。新的基于动物的模型促进了这种早期发现。

结论和相关性  使用新方法研究膀胱小细胞癌以了解新疗法的基因组景观的研究方向仍处于初步阶段。然而，此类研究正在成熟，可能会促进临床研究，从而有机会提高患有这种疾病的个体的生存率和与健康相关的生活质量。