Venetoclax and Azacitidine in Chinese patients with untreated acute myeloid leukemia ineligible for intensive chemotherapy

Acute Myeloid Leukemia (AML) patients are often found ineligible for aggressive standard chemotherapy in clinic because of old age and poor conditions. Current evidences suggest that targeted combination therapy might help these patients achieve a higher response rate with acceptable adverse events (AEs). 1 Venetoclax (VEN), a selective small-molecule BCL2 inhibitor, combined with hypomethylating agent (HMA), such as azacytidine (AZA), is now the standard treatment for older patients ineligible for intensive chemotherapy. 2 This combination therapy has proven to be effective in challenging AML population. 3 Recently, a subgroup analysis in VIALE-A trial 1 indicates that Chinese patients might not benefit more from a VEN plus AZA regimen compared with AZA alone. Unlike most other geographically defined populations in this same study, the patient number included in VIALE-A trial for Chinese population is relatively small. Due to a large population base with increasing AML patients in China, it would be important to further evaluate the effectiveness of such combo treatment in Chinese AML patients. Following a similar clinical design as the VIALE-A trial, this observational study was conducted to evaluate the efficacy and safety of VEN plus AZA combo compared with AZA alone in previously untreated Chinese AML patients who were ineligible for intensive induction chemotherapy. Further evidence was provided for associated genomic characteristics of Chinese AML patients who might benefit from such a combo treatment.