PMID- 37334144 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20230620 IS - 1178-6930 (Print) IS - 1178-6930 (Electronic) IS - 1178-6930 (Linking) VI - 16 DP - 2023 TI - Single-Center Retrospective Clinical Evaluation of Venetoclax Combined with HMAs and Half-Dose CAG for Unfit or Refractory/Relapsed AML. PG - 409-419 LID - 10.2147/OTT.S405611 [doi] AB - PURPOSE: The prognosis of patients with unfit or relapsed/refractory (R/R) AML remains poor. Venetoclax (VEN) has been shown to exhibit anti-leukemia stem cell activity; however, few studies have been published on the efficacy and safety of VEN combined with both hypomethylating agents (HMAs) and low-dose chemotherapy for patients with unfit or R/R AML. METHODS: This study retrospectively analyzed the clinical characteristics, treatment details, safety profile and clinical outcomes of patients with unfit or R/R AML treated with VEN+ HMAs+ half-dose CAG (LDAC, aclarubicin and granulocyte colony-stimulating factor). RESULTS: A total of 24 AML patients were involved in the study, of whom 13 (54.2%) were in the unfit group, and 11 (45.8%) were in the R/R group. FLT3 and IDH (8/24, 33.3%) were the most common gene aberrations. Patients in the R/R group were found to be more likely to carry KIT (5/11, 45.5%) compared with the unfit group (0/13, 0%) (P = 0.006). The ORR observed during the study was 83.3% (20/24; 14 CR, 2CRi, 4PR). In the unfit group, 11/13 (84.6%) patients achieved cCR (10 CR and 1 CRi); while 5/11 (45.5%) R/R patients achieved response (4 CR and 1 CRi). CR was observed in all AML patients with TP53 (5/5), GATA2 (3/3), CEBPA (3/3) and ASXL1 (3/3). The most common adverse events (AEs) during VEN+ HMAs+ half-dose CAG therapy were persistent cytopenias and infections. CONCLUSION: The results of this study confirm that VEN+ HMAs+ half-dose CAG is associated with promising efficacy (even high-risk molecular patterns) and tolerable safety profile in patients with unfit or R/R AML. Yet, the study involves only a small sample size, which should not be overlooked. As such, further studies on the efficacy of VEN combined with HMAs and half-dose CAG regimen in AML patients are essential. CI - (c) 2023 Chen et al. FAU - Chen, Xiaotong AU - Chen X AD - Department of Hematology, The First Affiliated Hospital of Harbin Medical University, Harbin, 150001, People's Republic of China. FAU - Zhao, Yanqiu AU - Zhao Y AD - Department of Hematology, The First Affiliated Hospital of Harbin Medical University, Harbin, 150001, People's Republic of China. FAU - Li, Qi AU - Li Q AD - Department of Hematology, The First Affiliated Hospital of Harbin Medical University, Harbin, 150001, People's Republic of China. FAU - Fan, Shengjin AU - Fan S AD - Department of Hematology, The First Affiliated Hospital of Harbin Medical University, Harbin, 150001, People's Republic of China. LA - eng PT - Journal Article DEP - 20230613 PL - New Zealand TA - Onco Targets Ther JT - OncoTargets and therapy JID - 101514322 PMC - PMC10276600 OTO - NOTNLM OT - BCL2 inhibitor OT - acute myeloid leukemia OT - combined chemotherapy OT - venetoclax COIS- There is no competing interest among the authors. EDAT- 2023/06/19 06:41 MHDA- 2023/06/19 06:42 PMCR- 2023/06/13 CRDT- 2023/06/19 03:01 PHST- 2023/02/03 00:00 [received] PHST- 2023/05/18 00:00 [accepted] PHST- 2023/06/19 06:42 [medline] PHST- 2023/06/19 06:41 [pubmed] PHST- 2023/06/19 03:01 [entrez] PHST- 2023/06/13 00:00 [pmc-release] AID - 405611 [pii] AID - 10.2147/OTT.S405611 [doi] PST - epublish SO - Onco Targets Ther. 2023 Jun 13;16:409-419. doi: 10.2147/OTT.S405611. eCollection 2023.