Evaluating the Efficacy of Venetoclax-Based Regimens in Relapsed/Refractory Acute Leukemia: A Real-World Study from Three Chinese Oncology Centers

Authors

Wei Zhang, Department of Hematology, Shanghai Cancer Center, Fudan University, Shanghai, China
Liyang Huang, Department of Oncology, West China Hospital, Sichuan University, Chengdu, China
Ming Zhao, Hematology Institute, Peking Union Medical College Hospital, Beijing, China
Rui Chen, Department of Clinical Pharmacy, West China School of Pharmacy, Sichuan University, Chengdu, China
Huiwen Liu, Department of Hematology, Shanghai Cancer Center, Fudan University, 270 Dong An Road, Xuhui District, Shanghai 200032, ChinaFollow

Article Type

Article

Abstract

Background: Relapsed or refractory (R/R) acute leukemia poses a significant therapeutic challenge, with limited options and poor prognosis. Venetoclax, a BCL-2 inhibitor, has shown promise in frontline acute myeloid leukemia (AML), but its real-world utility in R/R settings remains underexplored. Objective: To evaluate the efficacy and safety of venetoclax-based regimens in adult patients with R/R acute leukemia treated in routine clinical practice across three tertiary oncology centers in China. Methods: A retrospective multicenter study was conducted on 70 patients with R/R acute leukemia (AML or ALL) treated between January 2020 and December 2023. Patients received venetoclax combined with either hypomethylating agents (HMA group, n = 42) or low-dose cytarabine (LDAC group, n = 28). Response rates, overall survival (OS), event-free survival (EFS), and adverse events were analyzed. Kaplan-Meier survival estimates and Cox regression were used for outcome comparisons. Results: The complete response (CR/CRi) rate was significantly higher in the HMA + VEN group compared to the LDAC + VEN group (52.4% vs. 32.1%; p = 0.03). Median OS was 8.3 months (95% CI: 6.7--9.8) in the HMA cohort and 5.7 months (95% CI: 4.1--7.3) in the LDAC cohort (p = 0.04). Median EFS was 6.1 vs. 3.9 months, respectively (p = 0.02). Common grade ≥ 3 adverse events included febrile neutropenia (54.3%), thrombocytopenia (47.1%), and infections (32.9%). No early treatment-related deaths were observed. Conclusion: Venetoclax-based regimens, particularly in combination with hypomethylating agents, offer favorable response rates and survival benefits in patients with R/R acute leukemia. These findings support the integration of venetoclax into salvage protocols and warrant prospective validation in larger cohorts.

Keywords

Venetoclax, Acute myeloid leukemia, Relapsed leukemia, Refractory AML, Hypomethylating agents, Real-world study, China

Recommended Citation

Zhang, Wei; Huang, Liyang; Zhao, Ming; Chen, Rui; and Liu, Huiwen (2025) "Evaluating the Efficacy of Venetoclax-Based Regimens in Relapsed/Refractory Acute Leukemia: A Real-World Study from Three Chinese Oncology Centers," Muthanna Medical Journal: Vol. 12: Iss. 1, Article 1.
Available at: https://muthmj.mu.edu.iq/journal/vol12/iss1/1

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