Background:

Venetoclax (VEN) as a BCL-2 inhibitor, has shown promising results in treating adult acute myeloid leukemia (AML). However, studies on the relationship between VEN plasma concentrations and clinical efficacy and prognosis are still insufficient. According to the importance of plasma concentrations in clinical treatment, this study aims to analyze the impact of VEN plasma concentrations on the efficacy and prognosis of AML treatment.

Method:

This retrospective study included AML patients treated with VEN who were assessed for efficacy and at least one measurement of VEN plasma concentrations at West China Hospital from November 2021 to June 2024.

Results:

A total of 92 AML patients treated with VEN were included, encompassing both newly diagnosed and relapsed/refractory patients. In these patients, overall response rates (ORR), including CR, CRh, CRi, and PR, were 52.2%. Patients with response had higher VEN plasma concentrations compared with those no response (Wilcoxon, 2387.0 ng/ml vs. 1733.5 ng/ml, P=0.043) and had a significantly better overall survival (691d vs. 257d, P=0.014). There was a certain correlation between VEN plasma concentrations and efficacy (P=0.043). The optimal cut-off for VEN plasma concentrations was 2250 ng/ml and patients with higher VEN plasma concentrations (N=38) showed better response than lower concentrations (N=54) (chi-square, 65.8% vs. 42.6%, P=0.047). But there was no significant difference in survival on either side of 2250 ng/ml (358d vs. 268d, P=0.725).

Conclusion:

VEN plasma concentrations were associated with clinical response, suggesting that VEN plasma concentrations above 2250 ng/ml might achieve beneficial clinical outcomes. However, higher concentrations do not mean longer survival, which may be related to adverse reactions caused by high concentrations.

Disclosures

No relevant conflicts of interest to declare.

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2024
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