Background: Medical resources, especially blood products, were in short supply during the COVID-19. Less intensive therapy with hypomethylating agents/venetoclax (VEN) seems an effective treatment option for patients with acute myeloid leukemia (AML).
Objectives: To retrospectively analyze the efficacy and safety of VEN combined with azacitidine (AZA) in young adult patients with newly diagnosed (ND) AML.
Design: This was a retrospective study.
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, compared with a randomized historical study cohort that was administered intensive chemotherapy (IC) from January 2018 to December 2019.
Results: No rate of complete remission/complete remission with incomplete count recovery differences observed between the two arms reached statistical significance. Compared to traditional IC, minimal residual disease (MRD)-negative remission was achieved more quickly in patients treated with VEN + AZA regimens (after cycle 1: 8% in the IC group vs 56% in the VEN group, p = 0.0004; after cycle 2: 16% in the IC group vs 72% in the VEN group, p = 0.0001), especially in those AML patients who had a poor prognosis. The dependency of transfusion of red blood cell (RBC) and platelets during induction treatment was significantly lower in the VEN + AZA group (RBC: p = 0.0269; platelet: 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 = 0.0001; gastrointestinal side effects: 48% vs 12%, p = 0.0055). The total hospitalization cost of the VEN group was significantly less than that of the IC group (p = 0.0395).
Conclusion: In conclusion, our study indicated that VEN + AZA with a higher MRD-negative remission rate and less toxic appeared to be a therapy option for young patients with ND AML. However, further well-designed studies with larger numbers of patients are needed to confirm the benefits of VEN + AZA in this population.
Keywords: acute myeloid leukemia; azacitidine; venetoclax; young patients.
VEN + AZA in young newly diagnosed AML patients Study about a new treatment combination in young patients with newly diagnosed acute myeloid leukemia written by clinicians. Why was the study done? Acute myeloid leukemia (AML) is a hematological malignancy and the recommended standard induction regimen is intensive chemotherapy. A sufficient and safe blood supply is necessary for AML patients during the intensive chemotherapy. However, medical resources, especially blood products, were in short supply during COVID-19. Hence, doctors wanted to find a less intensive therapy with less blood products whereby preserving efficacy in this patient group. What did the researchers do? The research team collected medical records from patients who received this less intensive therapy and compared their clinical indicators with those of patients receiving intensive chemotherapy. What did the researchers find? A total of 50 people were included in the study, 25 in each group. There was no difference between the two groups in terms of complete remission rates. For those with a poor prognosis, the low-intensity regimen resulted in higher rates of remission and deeper remissions. Patients in the low-intensity therapy group had a lower incidence of adverse reactions than the intensive chemotherapy regimen. Patients in the low-intensity regimen group also had fewer numerical amounts of blood products than in the intensive chemotherapy regimen. What do the findings mean? This new treatment regimen seems to be a promising option with less intensive and less toxic for young untreated AML patients. More research is needed to support these findings.
© The Author(s), 2025.