Beat AML® Study Provides New Data on 14-Day vs. 28-Day Venetoclax-Azacitidine Treatment in AML

 Clinicians often reduce the duration of venetoclax because of side effects, but study finds 14-day schedule fell short in older patients with newly diagnosed AML

WASHINGTON, July 30, 2026 /PRNewswire/ -- New study results from Blood Cancer United's Beat AML® Master Clinical Trial, published in Blood provide new information about whether older patients with newly diagnosed acute myeloid leukemia (AML) can receive a shorter schedule of venetoclax when it's given with azacitidine.

The OPTI-AML study found that a 14-day venetoclax schedule did not meet criteria to show it performed similarly to the standard 28-day schedule based on complete remission rates. The findings also suggest that the optimal duration of venetoclax may vary by AML genetic mutation, reinforcing the need to tailor treatment based on disease biology.

Azacitidine plus venetoclax is standard treatment for older patients with newly diagnosed AML who are not eligible for intensive chemotherapy. While effective, the treatment can cause prolonged low blood counts, which may increase risk of infection and lead to treatment delays. In clinical practice, the duration of venetoclax is often reduced to 14 to 21 days to help patients recover blood counts, but evidence comparing shorter schedules has been limited and has not yet included a randomized trial, which is considered the gold standard in clinical research.

"The perception in the blood cancer community is that 28 days of venetoclax for two cycles is too toxic — it causes low counts, dose delays, and ultimately, clinicians may shorten the schedule," says lead study author Uma Borate, MBBS, MS, of The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) in Columbus. "Our study shows that shortening treatment to 14 days is not a one-size-fits-all solution for older patients with AML."

OPTI-AML, a sub-study of Beat AML, compared the standard 28-day venetoclax schedule with a 14-day schedule, both given with standard doses of azacitidine for two cycles. The study enrolled 169 patients ages 60 and older with newly diagnosed AML who were not eligible for intensive chemotherapy.

The study's primary endpoint was complete remission achieved at any time during the first two cycles of therapy. Complete remission rates were 49.4% for the 28-day schedule compared with 43% for the 14-day schedule. The 14-day schedule did not meet the study's prespecified criteria for noninferiority.

Findings also varied by genetic mutation. Patients with NPM1 or IDH2 mutations had higher complete remission rates with the 28-day schedule compared with the 14-day schedule, while patients without those mutations had comparable complete remission rates between the two arms. The results reinforce that AML is not a single disease, but many different diseases that may require treatment approaches tailored to disease biology.

"As Beat AML nears its 10-year milestone, this study shows how the trial continues to answer questions that matter in real-world care," said Lore Gruenbaum, Ph.D., chief scientific officer at Blood Cancer United. "We are not only looking for the next breakthrough — we are learning how to use today's treatments more effectively, safely and precisely for the patients most likely to benefit. These findings are an important reminder that reducing treatment intensity may help some patients, but it can also risk compromising response in others. Our goal is to generate the evidence clinicians need to make those decisions with greater confidence."

The findings are especially important as researchers continue to study new triplet combinations that build on azacitidine and venetoclax. The study authors noted that future research should continue evaluating how venetoclax duration can be optimized for different genetic subgroups, particularly as new targeted therapies are added to AML treatment.

About Blood Cancer United^® and the Beat AML^® Master Clinical Trial
Blood Cancer United (formerly The Leukemia & Lymphoma Society) is the largest global nonprofit focused on blood cancer patient support, research, and advocacy. The organization's mission is to cure blood cancer and improve the quality of life of all patients and their families. To achieve it, Blood Cancer United brings together a community of people—patients and their families, volunteers, healthcare providers, scientists, staff, partners, fundraisers, and philanthropists—who believe all blood cancer patients deserve longer, fuller lives. For support and to learn more, visit www.BloodCancerUnited.org.

The Beat AML® Master Clinical Trial (NCT03013998), launched in 2016 by Blood Cancer United, is the first collaborative precision medicine clinical trial in a blood cancer. The trial uses advanced genomic technology to match patients to the most promising targeted treatment based on their unique genetic mutations. The trial tests multiple therapies in multiple sub-studies simultaneously and generated strong results, showing superior survival rates and better quality of life for patients who were matched with therapies based on molecular testing. Over the past decade, Beat AML has grown into a powerful ecosystem of pharma companies, genetic testing experts, data specialists, regulatory agencies, and other industry partners working toward better and safer treatments for patients with AML. For more information, visit www.bloodcancerunited.org/beataml

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SOURCE Blood Cancer United® formerly The Leukemia & Lymphoma Society