Using Salvage Therapy in AML to Achieve Durable Remission

In acute myeloid leukemia (AML), immature cells called blasts multiply quickly and prevent healthy cells from functioning properly. The standard treatments for AML are chemotherapy combined with a targeted therapy and sometimes a stem cell transplant. 

What is a salvage strategy?

A salvage therapy is a second line of treatment used after previous treatments have failed. For AML that is relapsed or refractory (has come back or is resistant to treatment), different salvage strategies are being explored.

A recent study explored the benefits of different salvage therapies among people with relapsed or refractory AML.  

Genetic mutations in AML

Some genetic mutations, such as NPM1 and CBF, can make AML harder to treat. 

NPM1 Mutations. NPM1 is a gene that supports the development of a protein responsible for DNA repair and regulating cell function. Changes in the NPM1 gene lead to an uncontrolled growth of white blood cells. NPM1 Mutations are present in about 30-40% of adult AML cases and 2-8% of childhood AML cases.   

CBF Mutations. Core binding factor (CBF) AML is a subtype of AML that occurs when at least one of the following genetic abnormalities is present t(8;21) (q22;q22) or inv(16) (p13q22)/t(16;16). It occurs in about 12-15% of all cases of AML.  

What the research found about salvage therapy for AML

The salvage strategies used in the study were:  

• Stem cell transplantation
• Intensive chemotherapy
• Combination therapy with venetoclax and azacitidine (VEN-AZA)
• Other strategies 

For each of these strategies, researchers measured minimal residual disease (MRD) and overall survival (OS).

MRD refers to the number of cancer cells that remain in the body after treatment. MRD negativity occurs when there are no detectable cancer cells in the blood or bone marrow. It is associated with long-term survival and reduced risk of relapse. 

People who received intensive chemotherapy had the highest rate of MRD negativity (72%), followed by those who received VEN-AZA (33%), and other salvage therapies (17%).  

OS is a measure of how long a person lives after starting treatment, regardless of the cause of death. 

Overall survival was higher among people who received a stem cell transplant (82%) compared to those who did not (70%).   

Exploring outcomes among people with AML after salvage therapy will help open the door to new treatment strategies and help people achieve and maintain remission. 

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Sources: 

• Relationship between salvage strategy and outcomes in patients with CBF or NPM1- mutated AML 
• NIH-Salvage Therapy 
• Core binding factor acute myelogenous leukemia