Highlights from the 67th ASH Annual Meeting: Azacitidine+venetoclax outperforms standard care in AML

Patients newly diagnosed with acute myeloid leukaemia (AML) who are eligible for intensive chemotherapy fare significantly better with a combined regimen of azacitidine and venetoclax (AZA-VEN) compared with conventional induction chemotherapy, suggest study findings presented at ASH 2025.

AZA-VEN is the standard of care for older adults who are not fit enough for intensive chemotherapy. This trial is the first to test the superiority of this regimen to intensive induction chemotherapy, in fit patients.

“Our study met its primary endpoint, demonstrating that AZA-VEN improves event-free survival (EFS). It also leads to higher rates of overall response and composite complete response than intensive induction chemotherapy in younger, intensive chemotherapy-eligible patients,” said lead study author Amir Fathi, Director of the Center for Leukemia, Mass General Brigham Cancer Institute and Associate Professor of Medicine at Harvard Medical School, Boston, US.

“A greater proportion of patients successfully proceeded to transplant following response, with less early mortality, significantly improved quality of life during initial treatment, and less time in the hospital.”

In the trial, 172 adult patients were randomly assigned to receive either AZA-VEN or standard intensive induction chemotherapy. With a median follow-up of just under 22 months, EFS was significantly longer in the AZA-VEN arm. Median EFS was more than 14 months among those receiving AZA-VEN compared with a median of just over six months for those receiving induction chemotherapy. The effect of AZA-VEN remained protective even after adjusting for other variables.

At one year, 53 per cent of those in the AZA-VEN arm met criteria for EFS compared with 36 per cent in the control arm.

Participants receiving AZA-VEN experienced a higher overall response to treatment than those receiving induction chemotherapy, with 88 per cent of those in the AZA-VEN arm seeing an overall response and 78 per cent seeing a composite complete response, compared with 62 per cent and 54 per cent in the control arm, respectively.

They were also more likely to progress to a transplant, which occurred in 61 per cent of those receiving AZA-VEN and 40 per cent of those receiving induction chemotherapy.

The rate of grade 3 or 4 therapy-related adverse events was similar between study arms. Sixty-day mortality was zero in the AZA-VEN group and 5 per cent in the control group. Ten per cent of patients in the induction arm required admission to the intensive care unit during their index hospitalisation, compared with zero in the AZA-VEN arm.

The researchers plan to conduct further analyses to compare costs, the rate of infectious complications, and other factors that may inform treatment decisions for this patient population. In addition, they will assess the use of measurable residual disease status to provide key prognostic and predictive information across arms of the study and inform the optimal amount of treatment needed for AZA-VEN prior to transplant.