New Treatment Approach Could Redefine Standard Chemotherapy for Acute Myeloid Leukemia

For decades, people diagnosed with acute myeloid leukemia have faced an intensive treatment path that often begins with weeks in the hospital and powerful doses of chemotherapy. Now, research presented at the American Society of Hematology annual meeting suggests a less aggressive approach could be just as effective and possibly safer for many patients. 

Acute myeloid leukemia, or AML, is a fast-growing cancer of the bone marrow and blood that interferes with the body’s ability to produce healthy blood cells. While advances in cancer genetics have improved diagnosis and follow-up care, the mainstay “7+3” chemotherapy regimen has changed little since the 1970s. The treatment can cause severe short- and long-term side effects and is often too harsh for older or medically fragile adults. 

The new study tested whether combining two drugs, azacitidine and venetoclax, could match or surpass those results in a group of younger, fit patients who are traditionally considered strong enough to tolerate intensive chemotherapy. Researchers found the combination not only produced higher response rates but also led to fewer hospital stays, less time in intensive care and better quality of life during the first few weeks of receiving treatment. 

Promising Study Results 

The phase 2 clinical trial enrolled 172 adults across nine U.S. centers. Participants were randomly assigned to receive either the azacitidine-venetoclax combination or standard chemotherapy. 

After approximately 16 months of follow-up, 88% of patients in the azacitidine-venetoclax group responded to treatment, compared with 62% in the chemotherapy group. Event-free survival, which measures how long patients stay in remission without relapse or complications, was also higher with azacitidine-venetoclax, at 53% after one year versus 39% with chemotherapy. 

Participants receiving azacitidine-venetoclax required fewer days in the hospital, averaging about two weeks compared with five weeks for chemotherapy patients, and none experienced early treatment-related deaths. 

Making Leukemia Treatment More Tolerable 

Alison Walker, MD, a hematologist at Moffitt Cancer Center, said the data address one of the most important questions in the field, which is whether patients who qualify for intensive chemotherapy could safely and effectively be treated with azacitidine and venetoclax instead. 

Alison Walker, MD

“This study begins to answer a key question for physicians and patients: Can we use a less intensive treatment for newly diagnosed AML and achieve equal or better results?” Walker said. “The data suggest that for many patients, this may be possible.” 

She added that the traditional chemotherapy approach requires long hospital stays and carries risks of serious infection and even death during the first phase of treatment. 

“What’s encouraging here is that patients receiving azacitidine and venetoclax had fewer severe infections, spent less time in the hospital or ICU, and reported better quality of life early in treatment,” Walker said. “That’s significant progress for people navigating this disease.” 

Venetoclax belongs to a class of drugs called BCL-2 inhibitors, which work by triggering cancer cells to self-destruct. Azacitidine, a type of chemotherapy given as an injection or infusion, helps prevent cancer cells from growing and dividing. Together, the drugs target the disease at different points in its life cycle. 

A Potential Shift in Care 

AML affects about 20,000 Americans each year, most over age 60. Although survival rates have improved with advances in stem cell transplantation and targeted drugs, many adults still face limited options if they cannot tolerate intensive therapy. 

 Walker said ongoing studies will help determine whether azacitidine-venetoclax can extend overall survival compared with chemotherapy and whether it could eventually become the standard first-line treatment. 

“These results are a step toward lower-toxicity treatment for all patients with AML,” she said. “As more data become available, we’ll have a clearer understanding of how best to incorporate this approach into routine care, as there are unanswered questions regarding the effects on measurable residual disease and overall survival in the azacitidine-venetoclax treated patients.” 

Researchers plan to continue monitoring trial participants over time to assess durability of response and long-term survival. If confirmed, these findings could reshape expectations for how newly diagnosed leukemia is treated.