The objectives of this study are to evaluate the available literature regarding the oncologic effect of neoadjuvant and adjuvant chemotherapy in the treatment of patients with clinically non-metastatic upper tract urothelial carcinoma (UTUC) and locally advanced UTUC. We searched PubMed, Cochrane Library, and Scopus databases in November 2019, according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) statement. We included studies that compared patients with non-metastatic UTUC who received either neoadjuvant or adjuvant chemotherapy with patients who underwent surgery alone. Subgroup meta-analyses were also performed for studies that investigated only locally advanced UTUC. Overall, 36 studies were included in the review of which 22 studies and 15,378 patients were eligible for the meta-analysis. Neoadjuvant chemotherapy (NAC) was associated with higher rates of pathological downstaging (pDS) (RR 6.48, 95% CI 2.05–20.44, p = 0.001) and pathological complete response (RR 18.46, 95% CI 3.34–99.24, p = 0.001); and this was also proven in a subgroup analysis of studies that evaluated pDS in locally advanced UTUC (RR 3.18, 95% CI 2.0–5.07, p < 0.001). The association of NAC with overall survival (OS) and cancer-specific survival (CSS) was also statistically significant in all patients and in patients with locally advanced UTUC. Adjuvant chemotherapy (AC) was associated with improved metastasis-free survival (HR 0.65, 95% CI 0.55–0.76, p < 0.001) and CSS (HR 0.66, 95% CI 0.57–0.77, p < 0.001), which continued to be true for the patients with locally advanced UTUC. The association of AC with OS was only significant in patients with locally advanced UTUC. Perioperative chemotherapy might provide better survival outcomes in patients with clinically non-metastatic UTUC treated with radical nephroureterectomy. Neoadjuvant chemotherapy seems to have promising results, although high level of evidence is still lacking. Despite the low level, the body of evidence suggests a need for multimodal therapy of invasive UTUC.

本研究的目的是评估有关新辅助和辅助化疗在治疗临床非转移性上尿路尿路上皮癌 (UTUC) 和局部晚期 UTUC 患者中的肿瘤学效果的现有文献。根据系统评价和元分析的首选报告项目 (PRISMA) 声明，我们于 2019 年 11 月检索了 PubMed、Cochrane 图书馆和 Scopus 数据库。我们纳入了将接受新辅助或辅助化疗的非转移性 UTUC 患者与仅接受手术的患者进行比较的研究。还对仅调查局部晚期 UTUC 的研究进行了亚组荟萃分析。总体而言，该评价纳入了 36 项研究，其中 22 项研究和 15,378 名患者符合荟萃分析的条件。p  = 0.001）和病理完全反应（RR 18.46，95% CI 3.34–99.24，p  = 0.001）；并且在对局部晚期 UTUC 中评估 pDS 的研究的亚组分析中也证明了这一点（RR 3.18，95% CI 2.0–5.07，p  < 0.001）。NAC 与总生存期 (OS) 和癌症特异性生存期 (CSS) 的关联在所有患者和局部晚期 UTUC 患者中也具有统计学意义。辅助化疗 (AC) 与无转移生存期（HR  0.65，95 % CI 0.55–0.76，p < 0.001）和 CSS（HR 0.66，95% CI 0.57–0.77，p < 0.001)，对于局部晚期 UTUC 患者仍然如此。AC 与 OS 的关联仅在局部晚期 UTUC 患者中显着。对于接受根治性肾输尿管切除术治疗的临床非转移性 UTUC 患者，围手术期化疗可能会提供更好的生存结果。尽管仍缺乏高水平的证据，但新辅助化疗似乎有希望的结果。尽管水平较低，但大量证据表明需要对侵入性 UTUC 进行多模式治疗。