Venetoclax and Azcitidine for AML: Better Responses and Fewer Complications

The standard treatment for acute myeloid leukemia (AML) is intensive induction chemotherapy (IC). High doses of anti-cancer medications are given over several months. This intensive therapycan cause severe short-term and long-term side effects. Sometimes, because of a person's age or overall health, the risks of IC may outweigh the benefits. It is important to consider alternative treatments for those patients who may not be good candidates for IC.

Intensive chemotherapy is the standard first-line treatment for AML

For many younger people, the first-line treatment for AML is called a 7 + 3 regimen. A patient receives cytarabine for seven days. Cytarabine is a type of chemotherapy that works by targeting cancer cells that are dividing. During the first three days of cytarabine treatment, they will also receive short infusions of anthracycline. Anthracycline is a medication that damages the DNA of cancer cells. 

If remission is not achieved, patients typically receive another round of chemotherapy, called a reinduction, before they are able to move on to a stem cell transplant. 

One treatment that has been used as an alternative to IC is azacitidine (Vidaza, Celgene). Azacitidine is a nucleoside metabolic inhibitor that helps control disease and improve blood counts.  Research over the last few years has explored the benefits of combining azacitidine and venetoclax (Venclexta, AbbVie). Venetoclax , a targeted therapy known as a BCL-2 inhibitor that works by blocking the function of the protein that helps cancer cells to survive.

A recent study presented at the ASH 2025 Annual Meeting looked at differences in outcomes among patients with AML who received a combination of azacitidine and venetoclax (aza-ven) and others who received the standard of care intensive chemotherapy (IC). 

Outcomes were better for people who received aza-ven  

Event-free survival (EFS) refers to the timeframe after starting treatment that a patient lives without an adverse event or complication, such as: 

• Relapse 
• Return of certain symptoms
• Disease progression
• Secondary cancer
• Death

During a follow-up analysis 16 months after treatment, EFS was 53% for people who received aza-ven and 39% for people who received IC.

Overall response rate (ORR), a measure of the number of people who have a partial or complete response to treatment, was also higher at 88% for people who received aza-ven and 62% for those who received the standard IC.

Additionally, 61% of people who received aza-ven moved on to stem cell transplant compared to just 40% of people who received IC. 

Patients who received aza-ven also reported

• Better quality of life 

• Less symptom burden 

• Less depression

These outcomes help to improve our understanding of the benefits of different treatment approaches and support ongoing research to expand the treatment landscape for people with AML.

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