Venetoclax with intensive chemotherapy in younger patients with newly diagnosed acute myeloid leukemia

This study aimed to investigate the safety and effectiveness of venetoclax (Venclexta) combined with intensive chemotherapy in patients aged 65 or younger with acute myeloid leukemia (AML).   

This study concluded that venetoclax added to intensive chemotherapy was safe and effective in these patients.  

AML is the most common type of leukemia diagnosed in adults. Powerful chemotherapy such as cladribine (Leustatin), high-dose cytarabine (Cytosar-U), and idarubicin (Idamycin) or CLIA regimen is commonly used. However, many patients have a protein called BCL-2 in high quantities on leukemia cells. This leads to resistance to chemotherapy and poorer outcomes for these patients.

Venetoclax is a type of targeted therapy that works as a BCL-2 inhibitor. The addition of venetoclax to lower intensity chemotherapy has been shown to improve the outcomes of older and unfit patients with newly diagnosed acute myeloid leukemia (AML). However, whether adding venetoclax to intensive chemotherapy with CLIA in younger patients with AML patients is still unknown. 

This study involved 50 patients aged 18 to 65 years with a new diagnosis of AML, mixed phenotype acute leukemia, or high-risk myelodysplastic syndrome, who received no previous therapy for leukemia. All patients received CLIA chemotherapy with venetoclax added on days 2-8 of each course of chemotherapy. Patients were followed up for an average of 13.5 months and had an average of 1 course of treatment.   

94% of patients had a complete response (CR; no signs of leukemia left). 82% of patients had undetectable measurable residual disease (MRD). MRD means there were no cancer cells left after treatment. After 1 year, 74% of patients were still responding to treatment. 

After 1 year, 85% of patients were alive and 68% were alive with no complications from AML. 

The most common severe side effects were low white blood cells with fever (78%), high liver enzymes (14%), diarrhea (4%), and infections (6%). 

This study concluded that venetoclax added to intensive chemotherapy was safe and effective in younger patients with newly diagnosed AML

This study had a small number of participants and a very short follow-up period. Larger studies are needed.