Upper Tract Urothelial Carcinoma (UTUC)

Overview

Urothelial carcinoma arising in the renal pelvis or ureter; OncoTree code UTUC. Distinguished from bladder urothelial carcinoma by enrichment for FGFR3 alterations and a distinct hotspot distribution (PMID:37682528).

Cohorts in the corpus

Recurrent alterations

  • FGFR3 — oncogenic alterations in 43% (81/187) of localized UTUC, the highest frequency across urothelial disease states in the cohort (PMID:37682528).
  • FGFR3 R248C is enriched in upper-tract disease (22%, 18/81) versus bladder tumors (11%, 37/333; p=0.01) (PMID:37682528).
  • PIK3CA, TSC1, CDKN2A, CDKN2B, KDM6A — co-altered with FGFR3 in urothelial carcinoma broadly, including upper-tract cases (PMID:37682528).
  • Upper tract urothelial carcinoma shared FGFR3 alteration patterns with bladder urothelial carcinoma PMID:36543146
  • Targeted sequencing of upper-tract urothelial carcinoma identified recurrent FGFR3 mutations, copy number alterations, and shared genomic features with bladder urothelial carcinoma PMID:26278805

Subtypes

  • Localized UTUC shows the highest FGFR3 oncogenic alteration rate (43%) of any urothelial state examined, and a distinct R248C-enriched mutational profile (PMID:37682528).

Therapeutic landscape

  • erdafitinib — UTUC patients were included in the 32-patient real-world metastatic urothelial carcinoma treatment cohort (ORR 40%, median PFS 2.8 months) (PMID:37682528).
  • Primary/metastatic FGFR3 discordance in 26% (7/27) of paired cases argues for metastatic-site or cfDNA profiling to guide erdafitinib selection in UTUC (PMID:37682528).

Sources

  • PMID:37682528 — Guercio et al., Clinical Cancer Research 2023.

This page was processed by crosslinker on 2026-05-14. - PMID:36543146

This page was processed by crosslinker on 2026-05-14. - PMID:26278805

This page was processed by crosslinker on 2026-05-14.