Urothelial Cancer

P1, N=100, Recruiting, Zumutor Biologics Inc. | N=33 --> 100

P1/2, N=55, Recruiting, Merck Sharp & Dohme LLC

In preclinical patient-derived organoids (PDOs) and patient-derived xenograft (PDX) models, CDK4/6 inhibition plus immune checkpoint blockade demonstrates potent antitumor efficacy. Overall, our findings suggest this combination therapy as a promising therapeutic strategy for TPX2high and NEBC patients.

P=N/A, N=36, Not yet recruiting, Peking University First Hospital

P2, N=10, Recruiting, Henry Ford Health System | Not yet recruiting --> Recruiting | Trial completion date: Mar 2026 --> Jan 2028 | Initiation date: May 2025 --> Apr 2026 | Trial primary completion date: Dec 2025 --> Dec 2027

P2, N=20, Recruiting, Xiujuan Qu

Biomarkers predictive of treatment response-including PD-L1 expression and tumor mutational burden-are summarized, integrating recent clinical and translational evidence. We conclude by outlining future research directions focused on overcoming therapeutic resistance, refining predictive and prognostic biomarkers, and developing next-generation immunotherapies to improve clinical outcomes for patients.

These findings suggest that the two components are part of the same neoplastic process and, given the presence of urothelial carcinoma in situ, possibly represent the first reported example of urothelial carcinoma with divergent prostatic differentiation. Awareness of divergent differentiation in urothelial carcinomas has important diagnostic, prognostic and therapeutic implications and can be resolved with the use of ancillary molecular studies.

Accurate distinction of poorly differentiated prostate carcinoma from urothelial carcinoma requires correlation with prior diagnoses and a focused immunohistochemical panel, as misclassification carries significant therapeutic implications. Pathologists should maintain awareness of this uncommon mimic.

P3, N=590, Recruiting, Merck Sharp & Dohme LLC | Not yet recruiting --> Recruiting

Our hypothesis-generating findings suggest that dMMR/MSI-H may serve as a biomarker of sensitivity to single-agent ICIs in advanced UTUC. External validation in larger, ideally prospective, studies is needed to confirm the effectiveness and durability of immune checkpoint blockade in this molecular subgroup.

There was male predominance in urothelial bladder neoplasms. Most ages were after 50 years. The majority of tumors were high grade and already muscle invasive (at least pT2).