Table 2.

Clinical trials of pembrolizumab alone or in combination with chemotherapy or ADCs to treat urothelial cancer.

Study	Regimen	Inclusion Criteria	HR (mPFS)	ORR (months)	DoR (months)	mOS (months)	mPFS (months)	Citation
KEYNOTE-052 Phase II NCT02335424	Pembrolizumab	1 L la/mUC	N/A	24	NR (95% CI 9, NR)	N/A	N/A	[75]
KEYNOTE-052 Phase II NCT02335424 (long-term follow up)	Pembrolizumab	1 L la/mUC	N/A	28.6 (95% CI 24.1, 33.5)	30.1 (95% CI 18.1, NR)	11.3 (95% CI 9.7, 13.1)	2.2	[76]
KEYNOTE-045 Phase III NCT02256436 (two years follow-up)	Pembrolizumab vs. chemotherapy	2 L mUC	N/A	21.1 vs. 11.0%	Not reached vs. 4.4	10.3 vs. 7.4	2.1 vs. 3.3	[77]
KEYNOTE-057	Pembrolizumab	High-risk, non-muscle invasive UC	N/A	41 CR	N/A	N/A	N/A	[68]
KEYNOTE-361 Phase III NCT02853305	Pembrolizumab + chemo vs. pembrolizumab vs. chemo	1 L mUC	0.78 (95% CI 0.65, 0.93) (excludes monotherapy)	54.7 vs. 30.3 vs. 44.9	8.5 vs. 28.2 vs. 6.2	17.0 vs. 15.6 vs. 14.3	8.3 vs. 7.1 (excludes monotherapy)	[69]
Phase I/II NCT02437370	Pembrolizumab + either docetaxel or gemcitabine	2 L mUC	N/A	44 vs. 45	N/A	N/A	13.3 vs. 5.9	[78]
PEANUT Phase II NCT03464734	Pembrolizumab + nab-paclitaxel	≥2 L mUC	N/A	38.6 (95% CI 27, 51)	Not reached	Not reached (media follow-up 8.9)	5.9 (95% CI 3.1, 11.5)	[79]
Phase II NCT02581982	Pembrolizumab + paclitaxel	2 L mUC	N/A	33	N/A	11.7 months (95% CI 8.7, NR)	6 PFS 46.8	[80]
TROPHY U-01 cohort 3 Phase II NCT03547973	Pembrolizumab + sacituzumab govitecan	mUC after platinum	N/A	41 (95% CI, 26.3 to 57.9; 20% complete response rate)	11.1 (95% CI 4.8, NE)	12.7 (10.7, NE)	5.3 (95% CI 3.4–10.2)	[81]
RC48G001 Phase I/II NCT04879329	Disitamab vedotin +/− pembrolizumab	HER2+ 1 L mUC	Not recruiting	N/A	N/A	N/A	N/A	ClinicalTrials.gov
Phase III NCT05911295	Disitamab vedotin + pembrolizumab vs. chemotherapy	HER2+ 1 L la/mUC	Not recruiting	N/A	N/A	N/A	N/A	ClinicalTrials.gov

Abbreviation: Chemo. = chemotherapy; pembro. = pembrolizumab; N/A: not available; NR = not reached; NE = not estimable.