{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,16]],"date-time":"2026-01-16T02:22:02Z","timestamp":1768530122716,"version":"3.49.0"},"reference-count":40,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2023,11,20]],"date-time":"2023-11-20T00:00:00Z","timestamp":1700438400000},"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>Objective<\/jats:title><jats:p>This study aimed to evaluate the efficacy and safety of venetoclax in combination with hypomethylating agents and CAG (VEN-DCAG) regimens in patients with relapsed\/refractory acute myeloid leukemia (R\/R AML).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The treatment response was analyzed by retrospective methods in R\/R AML patients treated with the VEN-DCAG regimen at our institution. This included, but was not limited to, CR\/CRi (complete remission\/complete remission with incomplete hematologic recovery) rate, measurable residual disease (MRD) negative rate, and overall survival (OS).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>20 patients with R\/R AML were recruited, with a median age of 40 years (10-70), 11 of whom were male (55%), and a median follow-up of 10.4 months (0.7-21.8). The overall response rate (ORR) after receiving 1 course of VEN-DCAG was 90% (18\/20), with 17 (85%) CR\/CRi (10 MRD-CR), 1 (5%) PR, and 2 (10%) NR. Subsequently, 12 patients (7 MRD-CR, 4 MRD+CR, 1 NR) were treated with the VEN-DCAG regimen, and 3 MRD+CR patients turned negative, and 13 patients finally achieved MRD-CR. Among them, 7 patients were in the relapse group, all achieving CR\/CRi (6 MRD-CR), and 13 patients in the refractory group, with 10 CR\/CRi (7 MRD-CR). The ORR for patients in the relapse and refractory groups was 100% (7\/7) and 84.6% (11\/13), respectively. Further, all patients experienced adverse events (AEs) of varying degrees of severity, with hematologic AEs primarily consisting of myelosuppression, while non-hematologic AEs were more common in the form of fever, gastrointestinal distress, and infections. 11 patients were followed up with bridging allogeneic hematopoietic stem cell transplantation (allo-HSCT) therapy. At the last follow-up, 11 patients (7 MRD-CR, 4 MRD+CR) who received allo-HSCT, 1 (MRD+CR) died, and 9 patients (6 MRD-CR, 1 PR, 2 NR) who did not receive allo-HSCT, 5 (2 MRD-CR, 1 PR, 2 NR) died as well.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>The VEN-DCAG regimen may be an effective treatment option for R\/R AML patients, with high ORR and MRD negative remission rates in both the relapsed and refractory groups. It is recommend that patients should be bridged to allo-HSCT as soon as possible after induction to CR by the VEN-DCAG regimen, which can lead to a significant long-term survival benefit.<\/jats:p><\/jats:sec><jats:sec><jats:title>Clinical trial registration<\/jats:title><jats:p><jats:uri>https:\/\/www.chictr.org.cn\/<\/jats:uri>, identifier ChiCTR2300075985.<\/jats:p><\/jats:sec>","DOI":"10.3389\/fimmu.2023.1269163","type":"journal-article","created":{"date-parts":[[2023,11,20]],"date-time":"2023-11-20T04:25:45Z","timestamp":1700454345000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":5,"title":["Venetoclax combined with hypomethylating agents and the CAG regimen in relapsed\/refractory AML: a single-center clinical trial"],"prefix":"10.3389","volume":"14","author":[{"given":"Yifan","family":"Liu","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yanfen","family":"Li","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ran","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zhangyu","family":"Yu","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yu","family":"Jing","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1965","published-online":{"date-parts":[[2023,11,20]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.20.03736","article-title":"Venetoclax combined with FLAG-IDA induction and consolidation in newly diagnosed and relapsed or refractory acute myeloid 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