Efficacy and safety of venetoclax and azacitidine for acute myeloid leukemia in China: a real-world single-center study
Abstract Background Venetoclax (VEN) and azacitidine (AZA) are used to treat patients with newly diagnosed acute myeloid leukaemia (AML) who are unfit for intensive chemotherapy and those with relapsed or refractory AML. Understanding the real-world usage patterns and outcomes after VEN-AZA therapy...
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BMC
2025-06-01
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| Online Access: | https://doi.org/10.1186/s12885-025-14167-z |
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| author | Jie-Fei Bai Ting Wang Jiang-Tao Li Chun-Li Zhang Long Qian Ya-Zi Yang Xiao-Ya Yun Jing-Jing Yin Fei Zhao Wei-Dong Ding Bao-Li Xing Shang-Yong Ning Lei Pei Xiao-Dong Xu Hui Liu Ru Feng |
| author_facet | Jie-Fei Bai Ting Wang Jiang-Tao Li Chun-Li Zhang Long Qian Ya-Zi Yang Xiao-Ya Yun Jing-Jing Yin Fei Zhao Wei-Dong Ding Bao-Li Xing Shang-Yong Ning Lei Pei Xiao-Dong Xu Hui Liu Ru Feng |
| author_sort | Jie-Fei Bai |
| collection | DOAJ |
| description | Abstract Background Venetoclax (VEN) and azacitidine (AZA) are used to treat patients with newly diagnosed acute myeloid leukaemia (AML) who are unfit for intensive chemotherapy and those with relapsed or refractory AML. Understanding the real-world usage patterns and outcomes after VEN-AZA therapy failure is crucial, yet poorly studied. Methods This single-centre retrospective cohort study included 50 patients with AML (29 newly diagnosed and 21 relapsed or refractory cases) who were treated between January 2020 and November 2023. The primary endpoint was overall survival (OS), and secondary endpoints included composite complete remission (CR), partial remission (PR), overall response rate (ORR), event-free survival (EFS), minimal residual disease (MRD), adverse events (AEs), and post-VEN-AZA failure. Results Among the newly diagnosed patients (median age, 74 years; follow-up 10.1 months), the median EFS was 9.87 months (95% CI, 6.54–13.2 months) and OS was 11.93 months (95% CI, 7.6–16.29 months). The ORR was 85.7%, CR/CR with incomplete haematologic recovery (CRi) was achieved in 67.9% of patients, and MRD negativity was observed in 26.3% of the cohort. Post-treatment failure included VEN-AZA combined with gilteritinib, chidamide, or selinexor, which resulted in PR or CRi. The median OS after post-failure was 1.6 months. Among relapsed or refractory cases (median age 65 years; follow-up 8.53 months), median EFS was 5.2 months (95% CI, 1.8–8.6 months), and OS was 9.1 months (95% CI, 3.01–15.19 months). The ORR was 52.4%, CR/CRi was achieved in 42.9% of patients, and MRD negativity was observed in 11.11% of the cohort. Post-failure treatments include induction chemotherapy, VEN-AZA combined with enasidenib or gilteritinib, and participation in clinical trials, which yielded varying responses. The median OS after failure was 0.67 months, and the most common AEs were haematological and infectious complications. Conclusions VEN-AZA demonstrated high efficacy and manageable toxicity in patients with AML. Following VEN-AZA failure, the combination of VEN-AZA with targeted therapies has shown better efficacy than other VEN-AZA alone, whereas induction chemotherapy or clinical trials were preferred after second-line failure. Larger multicentre studies are warranted to validate these findings. |
| format | Article |
| id | doaj-art-50bf3e901cda44bfbbd251f49ea0c38f |
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| language | English |
| publishDate | 2025-06-01 |
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| spelling | doaj-art-50bf3e901cda44bfbbd251f49ea0c38f2025-08-20T02:05:41ZengBMCBMC Cancer1471-24072025-06-0125111310.1186/s12885-025-14167-zEfficacy and safety of venetoclax and azacitidine for acute myeloid leukemia in China: a real-world single-center studyJie-Fei Bai0Ting Wang1Jiang-Tao Li2Chun-Li Zhang3Long Qian4Ya-Zi Yang5Xiao-Ya Yun6Jing-Jing Yin7Fei Zhao8Wei-Dong Ding9Bao-Li Xing10Shang-Yong Ning11Lei Pei12Xiao-Dong Xu13Hui Liu14Ru Feng15Department of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesDepartment of Hematology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical SciencesAbstract Background Venetoclax (VEN) and azacitidine (AZA) are used to treat patients with newly diagnosed acute myeloid leukaemia (AML) who are unfit for intensive chemotherapy and those with relapsed or refractory AML. Understanding the real-world usage patterns and outcomes after VEN-AZA therapy failure is crucial, yet poorly studied. Methods This single-centre retrospective cohort study included 50 patients with AML (29 newly diagnosed and 21 relapsed or refractory cases) who were treated between January 2020 and November 2023. The primary endpoint was overall survival (OS), and secondary endpoints included composite complete remission (CR), partial remission (PR), overall response rate (ORR), event-free survival (EFS), minimal residual disease (MRD), adverse events (AEs), and post-VEN-AZA failure. Results Among the newly diagnosed patients (median age, 74 years; follow-up 10.1 months), the median EFS was 9.87 months (95% CI, 6.54–13.2 months) and OS was 11.93 months (95% CI, 7.6–16.29 months). The ORR was 85.7%, CR/CR with incomplete haematologic recovery (CRi) was achieved in 67.9% of patients, and MRD negativity was observed in 26.3% of the cohort. Post-treatment failure included VEN-AZA combined with gilteritinib, chidamide, or selinexor, which resulted in PR or CRi. The median OS after post-failure was 1.6 months. Among relapsed or refractory cases (median age 65 years; follow-up 8.53 months), median EFS was 5.2 months (95% CI, 1.8–8.6 months), and OS was 9.1 months (95% CI, 3.01–15.19 months). The ORR was 52.4%, CR/CRi was achieved in 42.9% of patients, and MRD negativity was observed in 11.11% of the cohort. Post-failure treatments include induction chemotherapy, VEN-AZA combined with enasidenib or gilteritinib, and participation in clinical trials, which yielded varying responses. The median OS after failure was 0.67 months, and the most common AEs were haematological and infectious complications. Conclusions VEN-AZA demonstrated high efficacy and manageable toxicity in patients with AML. Following VEN-AZA failure, the combination of VEN-AZA with targeted therapies has shown better efficacy than other VEN-AZA alone, whereas induction chemotherapy or clinical trials were preferred after second-line failure. Larger multicentre studies are warranted to validate these findings.https://doi.org/10.1186/s12885-025-14167-zAcute myeloid leukemiaHypomethylating agentsVenetoclaxRelapseRefractory |
| spellingShingle | Jie-Fei Bai Ting Wang Jiang-Tao Li Chun-Li Zhang Long Qian Ya-Zi Yang Xiao-Ya Yun Jing-Jing Yin Fei Zhao Wei-Dong Ding Bao-Li Xing Shang-Yong Ning Lei Pei Xiao-Dong Xu Hui Liu Ru Feng Efficacy and safety of venetoclax and azacitidine for acute myeloid leukemia in China: a real-world single-center study BMC Cancer Acute myeloid leukemia Hypomethylating agents Venetoclax Relapse Refractory |
| title | Efficacy and safety of venetoclax and azacitidine for acute myeloid leukemia in China: a real-world single-center study |
| title_full | Efficacy and safety of venetoclax and azacitidine for acute myeloid leukemia in China: a real-world single-center study |
| title_fullStr | Efficacy and safety of venetoclax and azacitidine for acute myeloid leukemia in China: a real-world single-center study |
| title_full_unstemmed | Efficacy and safety of venetoclax and azacitidine for acute myeloid leukemia in China: a real-world single-center study |
| title_short | Efficacy and safety of venetoclax and azacitidine for acute myeloid leukemia in China: a real-world single-center study |
| title_sort | efficacy and safety of venetoclax and azacitidine for acute myeloid leukemia in china a real world single center study |
| topic | Acute myeloid leukemia Hypomethylating agents Venetoclax Relapse Refractory |
| url | https://doi.org/10.1186/s12885-025-14167-z |
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