ArticleBMC cancer2025
Efficacy and safety of venetoclax and azacitidine for acute myeloid leukemia in China: a real-world single-center study.
Article in BMC cancer, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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Who cites it
5 citing papers in PubMed.
- Rosuvastatin enhances the efficacy of venetoclax-azacitidine in older acute myeloid leukemia patients via reducing T-cell exhaustion.Cancer immunology, immunotherapy : CII · 2026Trial
- Targeting the nuclear export receptor exportin-1 in acute myeloid leukaemia: From biology to clinical translation.Clinical and translational medicine · 2026Review
- The prognostic value of maintaining minimal residual disease status in patients with acute myeloid leukaemia receiving azacitidine-based therapy.BMC cancer · 2026Article
- Safety and efficacy of semi-dose venetoclax plus azacitidine in unfit acute myeloid leukemia patients in China: a real-world single-center study.Investigational new drugs · 2025Article
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16 authors.
Funding
Abstract
backgroundVenetoclax (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.
methodsThis 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.
resultsAmong 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.
conclusionsVEN-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.
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