{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Liu Q"],"funding":["Liping Dou"],"pagination":["e70734"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11891779"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["14(5)"],"pubmed_abstract":["<h4>Introduction</h4>Currently, there are only a few avaailable treatment options for patients with relapsed and refractory acute myeloid leukemia (R/R AML).<h4>Methods</h4>We conducted a single-center, phase 1 prospective study (ChiCTR2200065634) to evaluate the efficacy and safety of chidamide, demethylating drugs (azacitidine), cytarabine, aclacinomycin, and G-CSF plus venetoclax (CDCAG-VEN) in patients with R/R AML. The previous CDCAG regimen was used as a historical control to compare its efficacy and safety. Thirty and 22 patients received one course of CDCAG with or without a 14-day course of venetoclax, respectively.<h4>Results</h4>The overall response rate (ORR) was significantly higher in the CDCAG-VEN group than in the CDCAG-treated group (78.6% vs. 45.5%; p = 0.015), and the CDCAG-VEN group achieved a better trend of measurable residual disease-negative response (61.1% vs. 22.2%, p = 0.134). Compared with the CDCAG group, the CDCAG-VEN group exhibited significantly better 1-year overall survival (63.3% vs. 35.1%, p = 0.005) and progression-free survival (76.7% vs. 36.0%, p = 0.022). The duration of response was notably better in the CDCAG-VEN group than in the CDCAG group (71.2% vs. 34.3%, p = 0.021) and had a lower cumulative incidence of relapse (22.2% vs. 48.9%, p = 0.095). The neutrophil and platelet recovery times were similar between the CDCAG-VEN and CDCAG groups (neutrophil: 18 days vs. 19 days, p = 0.293; platelet: 18 days vs. 19 days, p = 0.311). The frequencies of adverse events were comparable between both groups, except for a lower incidence of thrombosis in the CDCAG-VEN group (0% vs. 22.7%, p = 0.006).<h4>Discussion</h4>In conclusion, venetoclax in combination with CDCAG is an effective and safe treatment regimen for R/R AML, thereby rapidly identifying chemosensitive patients and inducing measurable residual disease-negative remission in a high proportion of patients with R/R AML."],"journal":["Cancer medicine"],"pubmed_title":["Chidamide in Combination With DCAG With or Without Venetoclax for Relapsed/Refractory Acute Myeloid Leukemia."],"pmcid":["PMC11891779"],"funding_grant_id":["20230484407","82270162","24BJZ30","7222175","2021YFA1100904","21BJZ30","82200169","82270224","21WQ034","2023YFC2507800"],"pubmed_authors":["Xu L","Jing Y","Lv L","Dou L","Zhang X","Liu Q","Gao W","Wang L"],"additional_accession":[]},"is_claimable":false,"name":"Chidamide in Combination With DCAG With or Without Venetoclax for Relapsed/Refractory Acute Myeloid Leukemia.","description":"<h4>Introduction</h4>Currently, there are only a few avaailable treatment options for patients with relapsed and refractory acute myeloid leukemia (R/R AML).<h4>Methods</h4>We conducted a single-center, phase 1 prospective study (ChiCTR2200065634) to evaluate the efficacy and safety of chidamide, demethylating drugs (azacitidine), cytarabine, aclacinomycin, and G-CSF plus venetoclax (CDCAG-VEN) in patients with R/R AML. The previous CDCAG regimen was used as a historical control to compare its efficacy and safety. Thirty and 22 patients received one course of CDCAG with or without a 14-day course of venetoclax, respectively.<h4>Results</h4>The overall response rate (ORR) was significantly higher in the CDCAG-VEN group than in the CDCAG-treated group (78.6% vs. 45.5%; p = 0.015), and the CDCAG-VEN group achieved a better trend of measurable residual disease-negative response (61.1% vs. 22.2%, p = 0.134). Compared with the CDCAG group, the CDCAG-VEN group exhibited significantly better 1-year overall survival (63.3% vs. 35.1%, p = 0.005) and progression-free survival (76.7% vs. 36.0%, p = 0.022). The duration of response was notably better in the CDCAG-VEN group than in the CDCAG group (71.2% vs. 34.3%, p = 0.021) and had a lower cumulative incidence of relapse (22.2% vs. 48.9%, p = 0.095). The neutrophil and platelet recovery times were similar between the CDCAG-VEN and CDCAG groups (neutrophil: 18 days vs. 19 days, p = 0.293; platelet: 18 days vs. 19 days, p = 0.311). The frequencies of adverse events were comparable between both groups, except for a lower incidence of thrombosis in the CDCAG-VEN group (0% vs. 22.7%, p = 0.006).<h4>Discussion</h4>In conclusion, venetoclax in combination with CDCAG is an effective and safe treatment regimen for R/R AML, thereby rapidly identifying chemosensitive patients and inducing measurable residual disease-negative remission in a high proportion of patients with R/R AML.","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Mar","modification":"2025-04-04T08:22:50.351Z","creation":"2025-04-04T08:22:50.351Z"},"accession":"S-EPMC11891779","cross_references":{"pubmed":["40062510"],"doi":["10.1002/cam4.70734"]}}