In a nutshell
This study aimed to investigate the safety and effectiveness of venetoclax (Venclexta) combined with low dose cytarabine (LDAC; Cytosar-U) in patients with acute myeloid leukemia (AML) who cannot undergo intensive chemotherapy.
This study concluded that this combination is a promising treatment for these patients.
Some background
Effective treatment options are limited for patients with acute myeloid leukemia (AML) who cannot tolerate intensive chemotherapy. Cytarabine is a standard chemotherapy used for the treatment of AML. However, in high, standard doses, it can cause severe toxicity in frail patients. Venetoclax is a targeted therapy used to treat adults with AML and other types of leukemia. It blocks a protein leading to cancer cell death.
A previous small study showed that venetoclax combined with low-dose cytarabine (LDAC) improves the response rates in patients with AML compared to LDAC alone. However, the results need clarifying in a larger trial.
Methods & findings
This study involved 211 patients with newly diagnosed AML who were ineligible for intensive chemotherapy. 143 patients were treated with venetoclax and LDAC (Ven group). 68 patients were treated with a placebo and LDAC (placebo group). The average follow-up was 12 months.
The average overall survival was 7.2 months for the Ven group compared to 4.1 months for the placebo group. Venetoclax added to LDAC was associated with a 25% improved chance of survival compared to LDAC alone.
The complete remission (CR) and CR with incomplete blood count recovery rate was 48% for the Ven group compared to 13% for the placebo group.
32% of patients in the Ven group experienced fever and low white blood cells as a side effect compared to 29% of the placebo group. Serious side effects were reported in 66% of the Ven group and 62% of the placebo group.
The bottom line
This study concluded that venetoclax plus LDAC improves the outcomes of patients with AML who cannot tolerate intensive chemotherapy compared to LDAC alone, with a manageable safety profile.
The fine print
This study had a short follow-up period. This study was funded by AbbVie, the manufacturer of venetoclax.
Published By :
Blood
Date :
Mar 27, 2020
