Abstract
Background: Relapsed or refractory (R/R) acute leukemia poses a signicant 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 efcacy 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), eventfree 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 signicantly 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 benets in patients with R/R acute leukemia. These ndings support the integration of
venetoclax into salvage protocols and warrant prospective validation in larger cohorts.
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 efcacy 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), eventfree 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 signicantly 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 benets in patients with R/R acute leukemia. These ndings support the integration of
venetoclax into salvage protocols and warrant prospective validation in larger cohorts.
Keywords
acute myeloid leukemia
China
Hypomethylating agents
Real-world study
Refractory AML
Relapsed leukemia
Venetoclax