{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Apolo AB"],"funding":["Intramural NIH HHS","NCATS NIH HHS","NCI NIH HHS","National Cancer Institute Intramural Program","Cancer Therapy Evaluation Program","National Cancer Institute of the National Institutes of Health","Merck","Center for Cancer Research"],"pagination":["45-55"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11698643"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["392(1)"],"pubmed_abstract":["Background
Muscle-invasive urothelial carcinoma is an aggressive disease with high rates of relapse. Whether pembrolizumab as adjuvant therapy would be effective in patients with high-risk muscle-invasive urothelial carcinoma after radical surgery is unknown.Methods
In this phase 3 trial, we randomly assigned patients, in a 1:1 ratio, to receive pembrolizumab at a dose of 200 mg every 3 weeks for 1 year or to undergo observation. Randomization was stratified according to pathological stage, centrally tested programmed death ligand 1 (PD-L1) status, and previous neoadjuvant chemotherapy. The coprimary end points were disease-free survival and overall survival in the intention-to-treat population. We considered the trial to be successful if either disease-free survival or overall survival was significantly longer with pembrolizumab than with observation.Results
A total of 702 patients underwent randomization; 354 were assigned to receive pembrolizumab, and 348 were assigned to observation. As of July 5, 2024, the median duration of follow-up for disease-free survival was 44.8 months. The median disease-free survival was 29.6 months (95% confidence interval [CI], 20.0 to 40.7) with pembrolizumab and 14.2 months (95% CI, 11.0 to 20.2) with observation (hazard ratio for disease progression or death, 0.73; 95% CI, 0.59 to 0.90; two-sided P = 0.003). Grade 3 or higher adverse events (regardless of attribution) occurred in 50.6% of the patients in the pembrolizumab group and in 31.6% of the patients in the observation group.Conclusions
Among patients with high-risk muscle-invasive urothelial carcinoma after radical surgery, disease-free survival was significantly longer with adjuvant pembrolizumab than with observation. (Funded by the National Cancer Institute of the National Institutes of Health and others; Alliance A031501 AMBASSADOR ClinicalTrials.gov number, NCT03244384.)."],"journal":["The New England journal of medicine"],"pubmed_title":["Adjuvant Pembrolizumab versus Observation in Muscle-Invasive Urothelial Carcinoma."],"pmcid":["PMC11698643"],"funding_grant_id":["UG1 CA233247","UG1 CA233302","UG1CA233337","U10CA180882","P30 CA008748","UG1 CA233327","U10CA180868 (NRG)","UL1 TR001863","UG1 CA233290","UG1 CA233191","UG1CA233160","UG1CA233180","UG1 CA233196","UG1 CA233193","UG1 CA233270","NCI ZIA BC 011351","UG1CA239767","UG1 CA232760","UG1 CA233373","UG1 CA233253","UG1CA233327","UG1CA233247","UG1CA233302","UG1 CA233337","U10 CA180882","ZIA BC011351","UG1 CA233180","U10 CA180820","UG1CA233193","UG1CA233270","UG1CA233191","UG1CA233290","UG1CA233253","U10 CA180868","UG1CA233373","UG1 CA239767","UG1 CA233160","UG1CA233196","U10 CA180821","U10 CA180888","U10CA180820 (ECOG-ACRIN)","UG1CA232760","U10CA180888","U10CA180821"],"pubmed_authors":["Wen Y","Woods M","Simon N","Huang J","Kim WY","Grivas P","Parikh R","Watt C","Reichert ZR","Morris MJ","Ballman KV","Teo MY","Chatta G","Singh P","Tripathi A","Cole S","Srinivas S","Perez Burbano G","Sharon E","Geynisman DM","Cordes L","Rosenberg JE","Odegaard M","McGregor B","Niglio S","Halabi S","Hahn O","Apolo AB","Kim JW","Streicher H","Berg S","Bilen MA","Petrylak D","Tan A","Sonpavde G","Hoffman-Censits J","Ley L","Sweis RF","Mitchell C","Moon H"],"additional_accession":[]},"is_claimable":false,"name":"Adjuvant Pembrolizumab versus Observation in Muscle-Invasive Urothelial Carcinoma.","description":"Background
Muscle-invasive urothelial carcinoma is an aggressive disease with high rates of relapse. Whether pembrolizumab as adjuvant therapy would be effective in patients with high-risk muscle-invasive urothelial carcinoma after radical surgery is unknown.Methods
In this phase 3 trial, we randomly assigned patients, in a 1:1 ratio, to receive pembrolizumab at a dose of 200 mg every 3 weeks for 1 year or to undergo observation. Randomization was stratified according to pathological stage, centrally tested programmed death ligand 1 (PD-L1) status, and previous neoadjuvant chemotherapy. The coprimary end points were disease-free survival and overall survival in the intention-to-treat population. We considered the trial to be successful if either disease-free survival or overall survival was significantly longer with pembrolizumab than with observation.Results
A total of 702 patients underwent randomization; 354 were assigned to receive pembrolizumab, and 348 were assigned to observation. As of July 5, 2024, the median duration of follow-up for disease-free survival was 44.8 months. The median disease-free survival was 29.6 months (95% confidence interval [CI], 20.0 to 40.7) with pembrolizumab and 14.2 months (95% CI, 11.0 to 20.2) with observation (hazard ratio for disease progression or death, 0.73; 95% CI, 0.59 to 0.90; two-sided P = 0.003). Grade 3 or higher adverse events (regardless of attribution) occurred in 50.6% of the patients in the pembrolizumab group and in 31.6% of the patients in the observation group.Conclusions
Among patients with high-risk muscle-invasive urothelial carcinoma after radical surgery, disease-free survival was significantly longer with adjuvant pembrolizumab than with observation. (Funded by the National Cancer Institute of the National Institutes of Health and others; Alliance A031501 AMBASSADOR ClinicalTrials.gov number, NCT03244384.).","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Jan","modification":"2026-06-01T23:13:03.156Z","creation":"2026-05-23T03:08:06.552Z"},"accession":"S-EPMC11698643","cross_references":{"pubmed":["39282902"],"doi":["10.1056/NEJMoa2401726"]}}