Checkpoint Inhibitors Herald New Era in Urothelial Cancer

Petros Grivas, MD, PhD
Published: Wednesday, Jan 09, 2019
Petros Grivas, MD

Petros Grivas, MD
Bladder cancer is very common and can cause significant morbidity, mortality, emotional distress, and financial burden for patients, families, societies, and healthcare systems worldwide. The most common histologic type is urothelial carcinoma (UC), which can affect the renal pelvis, ureter, bladder (most common site), and urethra. Long-term survival rates for locally advanced (with extravesical and/ or node-positive disease) and metastatic disease remain dismal, with an overall survival (OS) of 9 to 15 months and 5-year OS rate of 5% for metastatic cancer, even with platinum-based chemotherapy, which is the standard of care. The entire disease spectrum requires the development of new agents that are feasible and can improve outcomes. In that context, immune checkpoint inhibitors (ICIs), with their favorable safety and antitumor activity profiles, have heralded a new era in UC treatment.

A plethora of clinical trials employing ICIs and targeted agents against related immune pathways are ongoing in diverse UC spaces and treatment settings. Below are updates in the development of ICIs across UC treatment settings; however, because data emergence is very dynamic and continuous in this research field, this is not a comprehensive list.

Nonmuscle-Invasive Bladder Cancer

The International Bladder Cancer Group has developed refined recommendations on definitions, endpoints, and clinical trial designs for nonmuscle-invasive bladder cancer (NMIBC) to support alignment and harmonization across clinical trials.9 These guidelines also provide a conceptual framework regarding the use of comparator arms and time points for assessment in the settings of Bacillus Calmette–Guérin (BCG)–naïve, BCG-relapsing, and BCG-unresponsive disease.
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