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Venetoclax: A Medication that is Changing the AML Landscape
Posted: Jul 08, 2022
Venetoclax: A Medication that is Changing the AML Landscape image

In November of 2018, Venetoclax was approved in combination with azacitidine, decitabine or low-dose cytarabine for the treatment of newly diagnosed AML in adults who are 75 years or older or who have comorbidities that prevent the use of intensive induction chemotherapy. This FDA approval has greatly expanded available treatment options to many people with AML. 

What is Venetoclax?

Venetoclax is a BCL-2 inhibitor. BCL-2 is a protein that is present in high amounts in AML cancer cells where it helps these cells survive longer in the body and makes them more resistant to chemotherapy. Taking a BCL-2 inhibitor blocks the action of the BCL-2 protein and causes cancer cell death which often slows down the progression of AML.

Common side effects of Venetoclax include low white blood cell count (specifically neutropenia), nausea, anemia, diarrhea, upper respiratory tract infections, fatigue and low platelet count (thrombocytopenia). 

Read about two exciting studies showing the effectiveness of Venetoclax in combination with other drugs:

In June of 2020, a phase III trial called the VIALE-A trial showed a clinically meaningful improvement in response rates and overall survival with venetoclax + azacitidine when compared to azacitidine alone in newly diagnosed elderly patients with AML who were ineligible for intensive chemotherapy. The participants of this study had not been previously treated for their AML. The venetoclax + azacitidine combination prolonged overall survival and increased the likelihood of remission, especially in important mutational subsets like TP53, IDH1 and IDH2.

Results at a median follow-up of 20.5 months:

  venetoclax + azacitidine azacitidine
median overall survival   14.7 months 9.6 months
incidence of complete remission 36.7% 17.9%


A phase III trial in 2020 looked at venetoclax in combination with low-dose cytarabine (LDAC). This combination led to greater improvements in remission and overall survival compared to LDAC alone in adults with newly diagnosed AML who were 75 years or older or who had other medical conditions that prevented the use of intensive chemotherapy. Thirty-eight percent of participants in this study had secondary AML, and 20% had received prior hypomethylating agent treatment. The addition of venetoclax to LDAC led to quicker responses compared to LDAC alone. The authors concluded that venetoclax plus LDAC demonstrated a clinically meaningful improvement in remission rate and overall survival vs LDAC alone, with a manageable safety profile. Results from this study confirm venetoclax plus LDAC as an important frontline treatment for AML patients unfit for intensive chemotherapy. 

  venetoclax + LDAC LDAC
median overall survival*   7.2 months 4.1 months
incidence of complete remission** 48% 13%

*not statistically significant **includes Complete remission (CR) plus CR with incomplete blood count recovery

Current Venetoclax Clinical Trials

There are many studies currently being conducted to further test Venetoclax in combination with various other medications. Here are a few that are going on now. Click the link to view more information about each specific trial using our AML Clinical Trial Finder:

And many, many others. As you can see, researchers are exploring lots of different possibilities with venetoclax to determine which combinations provide the best results for different subsets of people with AML. There is still much to learn, but it is an extremely exciting time in AML research as we move closer and closer towards a cure for all!

The author Katie Braswell

about the author
Katie Braswell

Katie joined HealthTree as the Community Director for AML in 2021. She is a registered dietitian who previously worked at the VA hospital in Dallas, Texas where she coached veterans with blood cancer on how to use nutrition to improve their treatment outcomes and minimize cancer-related side effects. Katie is passionate about health education and patient empowerment. In her spare time, she loves to experiment with new recipes in the kitchen, spend time running outdoors and travel to new places.

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