Outcome of patients with advanced upper tract urothelial carcinoma treated with immune checkpoint inhibitors: A systematic review and meta-analysis

Crit Rev Oncol Hematol. 2021 Mar:159:103241. doi: 10.1016/j.critrevonc.2021.103241. Epub 2021 Feb 2.


Background: Advanced upper tract urothelial carcinoma (UTUC) has different molecular and genetic features from the commonest carcinoma of the bladder, suggesting a possible different sensitivity to immune-checkpoint inhibitors (ICI).

Methods: A systematic review and meta-analysis of all relevant clinical studies including advanced UTUC patients treated with ICI was conducted according to PRISMA guidelines.

Results: Six prospective trials for a total 2537 patients, including 396 (15.6 %) with advanced UTUC, were eligible for the analysis. In UTUC patients, the pooled ORR was 21.2 % (95 % CI, 12.5 %-33.7 %); the risk of death was reduced by 24 % over the standard platinum-based chemotherapy, but this was not statistically significant (hazard ratio = 0.76; 95 % confidence interval, 0.41-1.40; p = 0.37, χ2 = 3.28, p = 0.07; I2 = 70 %).

Conclusions: The current evidence does not support a statistically significant effect from ICI over the standard treatment for advanced UTUC patients. Properly performed pre-planned subgroup analyses from randomized clinical trials are eagerly awaited.

Keywords: Immune checkpoint inhibitors; Immunogenicity; Renal pelvis; UTUC; Upper tract urothelial cancer; Urothelial cancer.

Publication types

  • Meta-Analysis
  • Review
  • Systematic Review

MeSH terms

  • Carcinoma, Transitional Cell* / drug therapy
  • Humans
  • Immune Checkpoint Inhibitors*
  • Prospective Studies


  • Immune Checkpoint Inhibitors