Abstract

Upper tract urothelial (transitional cell) carcinoma (UTUC) is a rare malignant disease occurring in roughly two people per 100 000 population. UTUCs comprise 5–10% of urothelial carcinomas overall. 1 Roupret M Babjuk M Comperat E et al. European Association of Urology Guidelines on upper urinary tract urothelial carcinoma: 2017 update. Eur Urol. 2018; 73: 111-122 Summary Full Text Full Text PDF PubMed Scopus (448) Google Scholar A paucity of dedicated high-level evidence has led to extrapolation from studies of urothelial bladder cancer to establish treatment recommendations. Although UTUC biology and clinical features overlap with those of urothelial bladder cancer, differences exist in gene alteration patterns (eg, FGFR3 and HRAS are more frequently altered in UTUC whereas TP53, RB1, and ERBB2 are less frequently mutated), clinical stage at presentation (higher stage at presentation, on average, for patients with UTUC), gender differences (a higher percentage of women get UTUC, although in terms of total numbers they are still in a minority compared with men), and stage-for-stage outcomes (prognosis is poorer for patients with UTUC). 2 Audenet F Isharwal S Cha EK et al. Clonal relatedness and mutational differences between upper tract and bladder urothelial carcinoma. Clin Cancer Res. 2019; 25: 967-976 Crossref PubMed Scopus (67) Google Scholar , 3 Winters BR De Sarkar N Arora S et al. Genomic distinctions between metastatic lower and upper tract urothelial carcinoma revealed through rapid autopsy. JCI Insight. 2019; 4e128728 Crossref Scopus (12) Google Scholar As with many rare disease settings, patients with UTUC are disenfranchised because dedicated research is challenging. Adjuvant chemotherapy in upper tract urothelial carcinoma (the POUT trial): a phase 3, open-label, randomised controlled trialGemcitabine–platinum combination chemotherapy initiated within 90 days after nephroureterectomy significantly improved disease-free survival in patients with locally advanced UTUC. Adjuvant platinum-based chemotherapy should be considered a new standard of care after nephroureterectomy for this patient population. Full-Text PDF Open Access

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