Site Editor

Rebecca Olin, MD, MS


Combination of Venetoclax and Hypomethylating Agent for Favorable-Risk AML

By: Kelly M. Hennessey, PhD
Posted: Wednesday, February 17, 2021

Front-line treatments for acute myeloid leukemia (AML) are often limited due to poor performance status and comorbidities. A combination regimen of venetoclax with a hypomethylating agent has shown promising response rates among treatment-naive older or frail patients along with patients who have relapsed or refractory disease. Ibrahim Aldoss, MD, of City of Hope Comprehensive Cancer Center, Duarte, California, and colleagues found that patients with newly diagnosed and relapsed or refractory favorable-risk AML experienced high rates of remission and durable responses with this combination therapy. The results of their study were published in the American Journal of Hematology.  

The study retrospectively analyzed the activity of venetoclax with a hypomethylating agent to identify response factors in patients with favorable-risk AML. A total of 46 patients received the combination therapy. Of the total, 57% (n = 26) had newly diagnosed AML and 43% (n = 20) had relapsed or refractory AML. The median patient age was 70; however, patients with relapsed or refractory AML were younger than those with a newly diagnosed disease (56 years vs. 72 years.) Favorable-risk AML alterations among the patient population included 10 patients (22%) with core-binding factor leukemia, 24 patients (52%) with NPM1 mutations, and 12 patients (26%) with bi-allelic CEBPA mutations.

Within the cohort, 24 patients (52%) experienced complete remission, and 13 patients (28%) achieved complete remission with incomplete hematologic recovery. The median overall survival for the cohort was 18 months.

“Our study suggests that venetoclax with hypomethylating agent therapy may represent a therapeutic alternative for older or unfit patients with favorable-risk disease,” suggested the researchers. “Given the safety profile and promising response, randomized prospective studies are warranted to compare venetoclax with [a] hypomethylating agent to standard intensive regimens in newly diagnosed, traditionally fit, older patients, who may be candidates for allogeneic hematopoietic cell transplantation,” they concluded.

Disclosure: For full disclosures of the study authors, visit

By continuing to browse this site you permit us and our partners to place identification cookies on your browser and agree to our use of cookies to identify you for marketing. Read our Privacy Policy to learn more.