Pathological and Oncological Sciences 1.
Bacsó, Dániel
Semmelweis University, Department of Urology; Centre for Translational Medicine
Dániel Bacsó1, Anikó Valikovics1, Gergely Agócs2, Péter Hegyi3, Isabel Pinto Amorim das Virgens4, Mahmoud Obeidat4, Gergely Bánfi1, Viktor Grünwald5, Laura S. Mertens6, Bas W.G. van Rhijn7, Péter Nyirady1, Tibor Szarvas8
1: Department of Urology, Semmelweis University, Budapest, Hungary; Centre for Translational Medicine, Semmelweis University, Budapest, Hungary
2: Centre for Translational Medicine, Semmelweis University, Budapest, Hungary; Institute for Translational Medicine, Medical School, University of Pécs, Pécs, Hungary
3: Centre for Translational Medicine, Semmelweis University, Budapest, Hungary; Institute for Translational Medicine, Medical School, University of Pécs, Pécs, Hungary; Institute of Pancreatic Diseases, Semmelweis University, Budapest, Hungary
4: Centre for Translational Medicine, Semmelweis University, Budapest, Hungary
5: Carolus Institute for Urologic Oncology, University of Duisburg-Essen, Essen, Germany
6: Department of Urology, Netherlands Cancer Institute, Amsterdam, The Netherlands
7: Department of Urology, Netherlands Cancer Institute, Amsterdam, The Netherlands; Department of Urology, Caritas St Josef Medical Center, University of Regensburg, Regensburg, Germany; Department of Urology, Leiden University Medical Center, Leiden, The Netherlands
8: Department of Urology, Semmelweis University, Budapest, Hungary; Carolus Institute for Urologic Oncology, University of Duisburg-Essen, Essen, Germany
Introduction: Urachal cancer (UrC) is a rare and aggressive malignancy lacking established systemic treatment guidelines. Platinum- and 5-fluorouracil (5-FU)–based regimens are most frequently used, while precision oncology and hyperthermic intraperitoneal chemotherapy (HIPEC) are emerging options for selected patients.
Aims: To assess and compare the efficacy of pharmacological therapies for advanced UrC.
Methods: A systematic literature search of PubMed, Embase, and the Cochrane Library was conducted on November 19, 2024 (CRD42024601810). Studies reporting systemic or pharmaceutical treatments for advanced UrC were included. Outcomes were objective response rate (ORR), disease control rate (DCR), and overall survival (OS). Pooled proportions were visualized using forest plots and bar charts. Individual patient data were used for Kaplan–Meier survival analyses.
Results: Seventy-four studies including 321 patients were analyzed. 5-FU–based regimens and 5-FU–platinum combinations achieved higher pooled ORRs (42% and 38%) compared with platinum-based therapies (19%). Among targeted therapies with ≥10 patients, EGFR inhibitors and immune checkpoint inhibitors demonstrated ORRs of 39% and 32%, respectively. FOLFOX showed the highest ORR (62%) among 5-FU–platinum regimens. Two-year OS was highest for 5-FU–platinum combinations (61%), followed by platinum-based (50%) and 5-FU–based therapies (43%), without statistically significant differences. HIPEC was associated with a pooled 5-year OS of 44%.
Conclusions: 5-FU-based therapies provided the highest ORR, and the combination of 5-FU and platinum proved to be associated with the longest OS. In contrast, platinum-based therapies were less effective according to ORR endpoints. Furthermore, targeted therapies also showed encouraging results.
Funding: This work was supported by the K139059 grant of the Ministry for Innovation and Technology from the source of the National Research Development and Innovation Fund. IPAV was supported by the EKÖP-2024 - the new national excellence program of the Ministry for Culture and Innovation, from the source of the National Research, Development and Innovation Office fund. The funders had no role in study design, data collection, data analysis, data interpretation, or the writing of the report.