Single-Center Experience of Venetoclax Combined with Azacitidine in Young Patients with Newly Diagnosed Acute Myeloid Leukemia
Authors: Xu Xuezhu , Liu Rui , Yang Ruoyu , Wang Fangxia , Zhao Wanhong , He Aili 
Published: December 2024

Abstract
Background
For younger newly diagnosed (ND) patients who are defined as those being &lt;60 years, the recommended standard induction regimen is intensive chemotherapy (IC). Although this regimen can achieve a high response rate, shortage in blood products as well as concerns about the safety of blood products have emerged and treatment options for ND AML patients are limited. The coronavirus 2019 (COVID-19) pandemic has imposed worldwide challenge and has negatively impacted blood product management. Given that the safety profile of VEN-based induction regimens was favorable, during the outbreak of COVID-19, we treated ND young patients (&lt;60 years) with VEN in combination with low-dose azacitidine (AZA). Then we assessed our single-institution experience with VEN+AZA, comparing outcomes with a historical study cohort that administered IC.
Aims
To retrospectively analyze the efficacy and safety of VEN + AZA in young adult patients with newly diagnosed acute myeloid leukemia (AML).
Methods
The clinical data of 25 AML patients treated with the VEN + AZA regimen from January 2021 to December 2023 at our center were collected, comparing with a historical study cohort that was administered IC during the period from January 2018 to December 2019.
Results
There was no difference in the distributions for gender, age, the FAB types, risk stratification and bone marrow blast count between the two groups. No rate of complete remission (CR) differences observed between the two arms reached statistical significance. Compared to traditional IC, MRD-negative remission was achieved more quickly in patients treated with VEN+AZA regimens (after cycle 1: 8% in IC group versus 56% in VEN+AZA group, P=0.0004; after cycle 2: 16% in IC group versus 72% in VEN group, P=0.0001). Surprisingly, we observed that AML patients with Intermediate or adverse risk in the VEN+AZA treatment group achieved higher MRD-negative rates compared with those in the IC treatment group (intermediate: 13% in 3+7 group vs 80% in VEN group, P=0.0149; adverse: 0% in 3+7 group vs 67% in VEN group, P= 0.0070), indicating that VEN+AZA treatment may be more effective and could achieve deeper remission in those AML patients who had a poor prognosis. The dependency of transfusion of red blood cells and platelets during induction treatment was significantly lower in VEN+AZA group (P = 0.0269; P = 0.0054). Compared with the standard IC, the incidence rate of non-hematological adverse events in VEN+AZA group was significantly decreased (infection: 100% vs 20 %, P = 0.001; gastrointestinal side effects: 48% vs 12%, P = 0.0055).
Conclusions
Our retrospective study showed that VEN + AZA could achieve higher CR rate and deeper remission in newly diagnosed AML with less transfusion need compared to IC in young patients with newly diagnosed AML. Especially, this regimen was effective in adverse risk patients. It deserved further well-designed large-sample study to validate its effectiveness and safety.