Therapeutic options for patients with non-muscle-invasive bladder cancer (NMIBC) have traditionally been limited to intravesical immunotherapy or chemotherapy. A considerable number of new options have been investigated in recent years. Our aim was to review the efficacy and toxicity of novel therapeutic options (results already reported or currently under investigation) for patients with NMIBC.
We assessed the efficacy of various novel therapeutic options by examining key endpoints in diverse settings, including recurrence, progression, overall survival, disease-specific survival, and complete response. We identified the principal advantages and limitations for each option. Safety was predominantly evaluated as the incidence of grade ≥3 adverse events. Our investigation focused on evidence from scientific articles and congress abstracts published in English within the past 5 yr.
To date, pembrolizumab, nadofaragene firadenovec, and the combination of BCG with N-803 have received US Food and Drug administration approval for the treatment of BCG-unresponsive carcinoma in situ of the bladder (with or without papillary tumours). Five phase 3 trials are recruiting BCG-naïve patients with high-risk NMIBC. There is increasing interest in an ablative rather than an adjuvant approach for patients with intermediate-risk NMIBC.
Novel drugs and device-assisted drug delivery systems are on the verge of changing the treatment of NMIBC. Novel intravesical options seem to have the same efficacy with fewer adverse events in comparison to systemic therapies.
We reviewed new therapy options for non-muscle-invasive bladder cancer. Two agents (pembrolizumab and nadofaragene firadenovec) have been approved to date. Ongoing trials are assessing direct delivery of drugs in solution into the bladder. This route seems to have similar efficacy and fewer side effects than intravenous immunotherapy.
European urology oncology. 2024 Jun 06 [Epub ahead of print]
Félix Guerrero-Ramos, Joost L Boormans, Siamak Daneshmand, Paolo Gontero, Ashish M Kamat, Morgan Rouprêt, Antoni Vilaseca, Shahrokh F Shariat
Department of Urology, Hospital Universitario 12 de Octubre, Madrid, Spain. Electronic address: ., Department of Urology, Erasmus University Medical Center, Rotterdam, The Netherlands., Department of Urology, University of Southern California/Norris Comprehensive Cancer Center, Los Angeles, CA, USA., Division of Urology, Molinette Hospital, University of Torino School of Medicine, Torino, Italy., Department of Urology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA., Department of Urology, Hôpital Pitié-Salpétrière, Paris, France., Department of Urology, Hospital Clínic, Barcelona, Spain; Department of Surgery and Surgical Specialties, University of Barcelona, Barcelona, Spain., Department of Urology, Comprehensive Cancer Center, Medical University of Vienna, Vienna, Austria; Department of Urology, University of Texas Southwestern Medical Center, Dallas, TX, USA; Institute for Urology and Reproductive Health, I.M. Sechenov First Moscow State Medical University, Moscow, Russia; Department of Urology, Second Faculty of Medicine, Charles University, Prague, Czech Republic; Department of Urology, Weill Cornell Medical College, New York, NY, USA; Division of Urology, Department of Special Surgery, Jordan University Hospital, The University of Jordan, Amman, Jordan.