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Adjuvant Pembrolizumab versus Observation in Muscle-Invasive Urothelial Carcinoma
Andrea B Apolo1, Karla V Ballman1, Guru Sonpavde1
1From the Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda (A.B.A., N.S., S.N., L.L., L.C.), the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore (J.H.-C.), and the Investigational Drug Branch, Cancer Therapy Evaluation Program, National Cancer Institute, National Institutes of Health, Rockville (H.S., E.S.) - all in Maryland; the Alliance Statistics and Data Management Center, Mayo Clinic, Rochester, MN (K.V.B., M.O., C.M., G.P.B.); AdventHealth Cancer Institute and the University of Central Florida, Orlando (G.S.); Dana-Farber/Harvard Cancer Center, Boston (S.B., B.M.); UNC Lineberger Comprehensive Cancer Center, Chapel Hill (W.Y.K.), and Duke University Medical Center and Duke Cancer Institute, Durham (J.H., S.H.) - both in North Carolina; the University of Kansas Cancer Center, Westwood (R.P.); Memorial Sloan Kettering Cancer Center, New York (M.Y.T., M.J.M., J.E.R.), and Roswell Park Comprehensive Cancer Center, Buffalo (G.C.) - both in New York; the University of Chicago Comprehensive Cancer Center, Chicago (R.F.S., C.W., Y.W., O.H.), and Loyola University Medical Center, Maywood (M.W.) - both in Illinois; the Alliance Protocol Operations Office, University of Chicago, Chicago (C.W., Y.W., O.H.); Fox Chase Cancer Center, Philadelphia (D.M.G.); Fred Hutchinson Cancer Center and the University of Washington, Seattle (P.G.); Rogel Cancer Center, University of Michigan, Ann Arbor (Z.R.R.); Yale Cancer Center, Yale School of Medicine, New Haven, CT (J.W.K., D.P.); Winship Cancer Institute, Emory University, Atlanta (M.A.B.); Mayo Clinic Comprehensive Cancer Center, Phoenix, AZ (P.S.); Oklahoma University Health Stephenson Cancer Center, Oklahoma City (A. Tripathi); University of Texas Southwestern Medical Center, Dallas (S.C.); Stanford University, Stanford (S.S.), and Kaiser Permanente Riverside Medical Center, Riverside (H.M.) - both in California; and Vanderbilt-Ingram Cancer Center, Nashville (A. Tan).
Adjuvant pembrolizumab significantly improved disease-free survival in patients with high-risk muscle-invasive urothelial carcinoma post-surgery. This immunotherapy offers a new standard of care for preventing disease recurrence.
Area of Science:
- Oncology
- Immunotherapy
- Urothelial Carcinoma Research
Background:
- Muscle-invasive urothelial carcinoma presents a high risk of relapse.
- The efficacy of adjuvant pembrolizumab in high-risk patients post-surgery remains unestablished.
Purpose of the Study:
- To evaluate the effectiveness of adjuvant pembrolizumab versus observation in patients with high-risk muscle-invasive urothelial carcinoma after radical surgery.
Main Methods:
- A phase 3 randomized trial assigned 702 patients 1:1 to pembrolizumab (200 mg every 3 weeks for 1 year) or observation.
- Stratification included pathological stage, PD-L1 status, and neoadjuvant chemotherapy.
- Coprimary endpoints were disease-free survival and overall survival.
Main Results:
- Pembrolizumab demonstrated a significant improvement in median disease-free survival (29.6 months vs. 14.2 months).
- The hazard ratio for disease progression or death was 0.73 (95% CI, 0.59 to 0.90; P=0.003).
- Grade 3+ adverse events occurred in 50.6% of the pembrolizumab group versus 31.6% in the observation group.
Conclusions:
- Adjuvant pembrolizumab significantly enhances disease-free survival in high-risk muscle-invasive urothelial carcinoma patients post-radical surgery.
- Pembrolizumab represents a promising therapeutic option for this patient population.
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