Favorable pathological response with enfortumab vedotin plus toripalimab as neoadjuvant therapy for HER2-negative muscle-invasive bladder cancer: a case report

PURPOSE: To report the efficacy and safety of enfortumab vedotin combined with toripalimab as neoadjuvant therapy in an elderly patient with muscle-invasive bladder cancer (MIBC) who sought bladder preservation. METHODS: An 83-year-old male presented with painless gross hematuria. Imaging revealed a 44 mm × 39 mm × 60 mm bladder mass, and biopsy confirmed high-grade invasive papillary urothelial carcinoma (cT2NxM0). After informed consent, the patient received three cycles of neoadjuvant therapy combining enfortumab vedotin (60 mg on days 1 and 8) with toripalimab (240 mg on days 1 and 8) in 21-day cycles. RESULTS: Treatment-related adverse events included hyperglycemia and rash, both managed conservatively without treatment interruption. Post-treatment MRI demonstrated marked tumor reduction, showing only bladder wall thickening. Subsequent transurethral resection of bladder tumor (TURBT) revealed chronic mucosal inflammation with histiocyte aggregation and focal cystic cystitis, with no residual carcinoma identified in the resected specimen, indicating a favorable pathological response. CONCLUSION: In this carefully selected elderly MIBC patient, neoadjuvant enfortumab vedotin combined with toripalimab was well tolerated and associated with marked tumor regression and a favorable pathological response on post-treatment TURBT. Bladder preservation was attempted, illustrating the feasibility of this regimen and generating a hypothesis for future investigation.