[Efficacy and Safety of Venetoclax-Based Chemotherapy Regimens for High-Risk Acute Myeloid Leukemia].

OBJECTIVE

To evaluate the efficacy and safety of venetoclax (VEN) in combination with hypomethylating agents and/or low-dose cytarabine (LDAC) in patients with relapsed or refractory (RR) or persistently positive minimal residual disease (MRD) acute myeloid leukemia (AML).

METHODS

RR or MRD persistently positive AML patients who received at least one cycle of VEN combined with hypomethylating agents (azacitidine/decitabine) and/or LDAC in the Fifth Medical Center of Chinese PLA General Hospital were retrospectively collected. The objective response rate (ORR), MRD negative conversion rate, relapse-free survival (RFS), overall survival (OS), and adverse events were observed, and efficacy and safety were analyzed.

RESULTS

A total of 29 patients were included in this study, including 24 cases with RR AML and 5 cases with persistently positive MRD. In the RR AML group, the median age was 56 (16-74) years old, 13 patients achieved CR/CRi, and the MRD of 12 patients turned negative after treatment. In this group, the median follow-up time was 28.6 months, the median overall survival (OS) time was 11.9 months and the median relapse-free survival (RFS) time was 27.7 months. The 1-year cumulative OS rate was 48.1%, and the 1-year cumulative RFS rate was 61.5%. The most common adverse events were neutropenia and anemia, both of which occurred in 21 patients. In the group of MRD persistently positive AML, the median age was 49 (17-66) years old. Among them, the MRD of 4 patients turned negative. The median follow-up time was 31.4 months, and both the median OS and RFS time were not reached. The 1-year cumulative OS rate and 1-year cumulative RFS rate were both 80.0%. The most common adverse events were neutropenia, thrombocytopenia, and hypokalemia, all of which occurred in 2 patients. No death-related adverse events occurred.

CONCLUSION

VEN combined with hypomethylating agents and/or LDAC shows good efficacy and safety in RR and MRD persistently positive AML patients, which provides a new possible treatment option for clinical practice.