What is the usefulness of measurable residual disease in patients with acute myeloid leukemia treated with venetoclax and azacitidine?

This study aimed to investigate the outcomes for acute myeloid leukemia (AML) patients treated with venetoclax (Venclexta) and azacitidine (Vidaza) who achieved complete remission (CR) and minimal measurable residual disease (MRD) after treatment. 

This study concluded that patients who achieved both CR and minimal MRD had improved outcomes compared to patients with responded but had measurable MRD.  

Venetoclax and azacitidine is a treatment combination used for the treatment of AML. Many patients achieve CR (the bone marrow and blood cell counts return to normal) after this treatment. However, after treatment, there may be a very small number of cancer cells left in the body. This is called MRD. Detectable MRD (more than 0.001 cancer cells left) is the reason for cancer relapse. 

In patients with AML treated with intense therapy, negative MRD (less than 0.001 cancer cells left) has been shown to be associated with better outcomes. However, it is not known whether negative MRD is also important in determining the outcomes of patients with AML treated with venetoclax and azacitidine. 

This study involved 190 patients with AML treated with venetoclax and azacitidine who achieved CR. 164 of these patients (86%) were evaluated for MRD. Patients were followed up for an average of 20.8 and 22.1 months. 

Negative MRD was achieved by 41% of patients. In patients with a CR and negative MRD, the average duration of remission (DOR), event-free survival (EFS; complications due to AML), and overall survival (OS) were not reached (were longer than the follow-up period). The 12-month estimate for DoR in this group was 81.2%. The 12-month estimate for EFS was 83.2% and for OS was 94%. 

In patients with a CR and detectable MRD, the average DoR was 9.7 months. The average EFS was 10.6 months and the average OS was 18.7 months.  

This study concluded that patients with AML who achieved a CR and negative MRD treated venetoclax and azacitidine had improved outcomes compared to patients with a CR and positive MRD.

The number of patients was rather small. MRD was measured at specific time points and was not continuous.