biostudies-literatureLiu QLiping Doue70734https://www.ebi.ac.uk/biostudies/studies/S-EPMC11891779biostudies-literatureUnknown14(5)<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.Cancer medicineChidamide in Combination With DCAG With or Without Venetoclax for Relapsed/Refractory Acute Myeloid Leukemia.PMC11891779202304844078227016224BJZ3072221752021YFA110090421BJZ30822001698227022421WQ0342023YFC2507800Xu LJing YLv LDou LZhang XLiu QGao WWang LfalseChidamide in Combination With DCAG With or Without Venetoclax for Relapsed/Refractory Acute Myeloid Leukemia.<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.2025-01-01T00:00:00Z2025 Mar2025-04-04T08:22:50.351Z2025-04-04T08:22:50.351ZS-EPMC118917794006251010.1002/cam4.70734