Single‑center analysis of a real‑world cohort of patients with metastatic urothelial carcinoma evaluated by NGS: molecular landscape and efcacy of targeted therapies
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César Gutiérrez Pérez [1] ; Enrique Lastra Aras [1] ; Patricia Saiz López [1] ; Enrique García Toro [1] ; Carmen Blanco Abad [1] ; Inmaculada Rodríguez Ledesma [1] ; María Pumares González [1] ; Miriam Vela Domínguez [1] ; Noelia Espinosa Cabria [1] ; Guillermo Crespo Herrero [1]
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[1] Complejo Asistencial Universitario de Burgos
Complejo Asistencial Universitario de Burgos
Burgos, España
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- Localización: Clinical & translational oncology, ISSN 1699-048X, Vol. 27, Nº. 3, 2025, págs. 1211-1220
- Idioma: inglés
- Texto completo no disponible (Saber más ...)
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Resumen
Purpose To describe the molecular profle of a real-world cohort of patients with metastatic urothelial carcinoma (mUC) and to evaluate the beneft of next-generation sequencing (NGS) panels in guiding therapy in patients with mUC and the outcomes of DNA-matched treatments recommended by a multidisciplinary molecular tumor board (MMTB).
Methods This was a single-center analysis of a real-world cohort of adult patients with mUC included in an ongoing trial that aimed to evaluate the clinical utility of NGS for solid tumors. Genomic analysis was performed for each patient, most of them using the Ion Torrent Oncomine Focus Assay. Genomic results were discussed during MMTB meetings.
Results We included 43 patients with mUC treated with platinum-based combinations and immunotherapy. Twenty-fve patients (58.1%; 95% CI 43.4–72.9) had at least one tumor pathogenic alteration. The MMTB classifed 16 (48.5%) of the 33 tumor pathogenic alterations found in our real-world cohort of mUC patients as ESCAT I, which is the maximum grade of actionability. After excluding patients who were not candidates for targeted therapies, the MMTB provided guidance on matched therapy for seven patients. Among these patients, three achieved a partial response for an overall response rate of 42.9%, a median progression-free survival of 7.3 months (95% CI 6.7–7.9) and a median overall survival of 10.9 months (95% CI 2.4–19.5).
Conclusions We recommend that all patients with mUC undergo NGS at diagnosis given the high percentage of patients with pathogenic alterations in our real-world cohort and the efcacy data of patients treated with targeted therapies.
