{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T21:19:54Z","timestamp":1790630394363,"version":"4.1.0"},"reference-count":72,"publisher":"MDPI AG","issue":"17","license":[{"start":{"date-parts":[[2021,8,27]],"date-time":"2021-08-27T00:00:00Z","timestamp":1630022400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cancers"],"abstract":"<jats:p>Upper tract urothelial carcinoma (UTUC) represents a rare and aggressive malignancy arising from the renal pelvis or ureter. It can develop sporadically or have a hereditary origin, such as Lynch syndrome, caused by DNA mismatch repair deficiency, leading to microsatellite instability phenotype. According to molecular characterization studies, UTUC presents different mutational profiles as compared to urinary bladder urothelial carcinomas. In particular, it has been reported that UTUC harbored a higher level of FGFR3 alterations associated with a T-cell depleted immune microenvironment. The therapeutic landscape in urothelial carcinoma is rapidly evolving, with immune checkpoint inhibitors forming part of the standard of care. A greater understanding of the molecular alterations and immune microenvironment leads to the development of new treatment combinations and targeted therapy. This review summarizes the available evidence concerning the use of immune checkpoint inhibitors and the biological rationale underlying their use in high-grade UTUC.<\/jats:p>","DOI":"10.3390\/cancers13174341","type":"journal-article","created":{"date-parts":[[2021,8,27]],"date-time":"2021-08-27T09:53:23Z","timestamp":1630058003000},"page":"4341","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":15,"title":["Efficacy of Immune Checkpoint Inhibitors in Upper Tract Urothelial Carcinomas: Current Knowledge and Future Directions"],"prefix":"10.3390","volume":"13","author":[{"given":"Jonathan","family":"Thouvenin","sequence":"first","affiliation":[{"name":"Oncology Department, Institut de Canc\u00e9rologie Strasbourg (ICANS), 67200 Strasbourg, France"},{"name":"Oncology Department, Institut Jules Bordet, 1000 Bruxelles, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nieves","family":"Mart\u00ednez Chanz\u00e1","sequence":"additional","affiliation":[{"name":"Oncology Department, Institut Jules Bordet, 1000 Bruxelles, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9658-2206","authenticated-orcid":false,"given":"Omar","family":"Alhalabi","sequence":"additional","affiliation":[{"name":"Genitourinary Medical Oncology Department, The University of Texas MD Anderson Cancer Center (MDACC), Houston, TX 77030, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Herv\u00e9","family":"Lang","sequence":"additional","affiliation":[{"name":"Urology Department, Strasbourg University Hospital, Nouvel H\u00f4pital Civil, 67000 Strasbourg, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nizar M.","family":"Tannir","sequence":"additional","affiliation":[{"name":"Genitourinary Medical Oncology Department, The University of Texas MD Anderson Cancer Center (MDACC), Houston, TX 77030, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philippe","family":"Barth\u00e9l\u00e9my","sequence":"additional","affiliation":[{"name":"Oncology Department, Institut de Canc\u00e9rologie Strasbourg (ICANS), 67200 Strasbourg, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabriel G.","family":"Malouf","sequence":"additional","affiliation":[{"name":"Oncology Department, Institut de Canc\u00e9rologie Strasbourg (ICANS), 67200 Strasbourg, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2021,8,27]]},"reference":[{"key":"ref_1","first-page":"62","article-title":"European association of urology guidelines on upper urinary tract urothelial carcinoma: 2020 update","volume":"79","author":"Babjuk","year":"2020","journal-title":"Eur. 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