{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,24]],"date-time":"2026-06-24T17:06:04Z","timestamp":1782320764459,"version":"3.54.5"},"reference-count":32,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2025,4,17]],"date-time":"2025-04-17T00:00:00Z","timestamp":1744848000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Immunol."],"abstract":"<jats:sec><jats:title>Introduction<\/jats:title><jats:p>Anti-PD-1 monotherapy has shown limited clinical efficacy in patients with relapsed\/refractory acute myeloid leukemia (r\/r AML). Our study aimed to analyze the effectiveness and safety of combining tislelizumab with a hypomethylating agent (HMA) plus CAG regimen in treating patients with r\/r AML, with an increased sample size and in comparison, with a historical control group for more reliable data support (ClinicalTrials.gov identifier NCT04541277).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The study included a total of 37 patients with r\/r AML who received the tislelizumab + HMA + CAG regimen.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The overall response rate was 69.4%, with a median overall survival of 12.1 months and event-free survival of 6.2 months. Multivariate analysis revealed that patients aged 40 or above exhibited a higher response rate, while those with lower leukemia burden (bone marrow blast percentage &amp;lt;40%) demonstrated improved overall survival and event-free survival. Additionally, bridging allogeneic hematopoietic stem cell transplantation was associated with extended event-free survival. Grade 2-3 immune-related adverse events were observed in 8.5% of patients, and no deaths were directly attributed to these events. After propensity score matching, the inclusion of tislelizumab appeared to positively influence the overall response rate and event-free survival compared to historical controls treated with HMA + CAG regimen.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>Overall, the combination regimen improved response rates while maintaining low incidence and severity of immune-related adverse events for r\/r AML patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Clinical trial registration<\/jats:title><jats:p><jats:uri>https:\/\/clinicaltrials.gov\/<\/jats:uri>, identifier NCT04541277.<\/jats:p><\/jats:sec>","DOI":"10.3389\/fimmu.2025.1533467","type":"journal-article","created":{"date-parts":[[2025,4,17]],"date-time":"2025-04-17T05:25:06Z","timestamp":1744867506000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":3,"title":["Updates from a single-center phase 2 study of PD-1 inhibitor combined with 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