Prognostic Factors in Patients with Acute Myeloid Leukemia Treated with the Combination of Venetoclax plus Azacitidine(VEN+AZA).

Objectives: To analyze the efficacy, safety, prognostic factors, factors affecting treatment continuation, suitable treatment candidates, and optimal administration schedule in patients with acute myeloid leukemia(AML)treated with venetoclax plus azacitidine(VEN+AZA).

Methods: We performed a retrospective analysis of the data of 39 patients with untreated or relapsed/refractory AML.

Results: The median duration of follow-up was 6 months, and the median number of treatment cycles was 2. The composite complete remission(CRc)achievement rate(complete remission+complete remission with incomplete hematological recovery)was 61.5%. The treatment discontinuation rate was 76.9%, the median overall survival (OS)was 7.7 months, and event-free survival(EFS)was 4.8 months. In subgroup analyses, significant differences in the OS were observed between subgroups stratified according to the cytogenetic risk, CRc achievement rate, and Charlson comorbidity index(CCI)(≤7 vs <7). A significant difference in the EFS was also observed between subgroups stratified according to the cytogenetic risk and CRc achievement rate. The response rate tended to be lower in the adverse cytogenetic risk subgroup. Patients who received VEN for 21 days or less in the first treatment cycle tended to have a better OS.

Conclusions: A lower OS and EFS were associated with a higher treatment discontinuation rate, lower number of treatment cycles, and lower CRc achievement rate than those observed in the VIALE-A trial. We considered that treatment continuation was important to improve the prognosis. We also concluded that it is important to select candidates suitable for VEN+AZA treatment and to modify the administration schedule.