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Frontline Therapy of AML in the Fit and Younger Population – Incorporating Molecularly Targeted Agents

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Mar 14, 2025, 18:27

Frontline Therapy of AML in the Fit and Younger Population – Incorporating Molecularly Targeted Agents

A paper by Keith Pratz and Harry Erba was published in American Journal of Hematology, titled:

“Frontline Therapy of AML in the Fit and Younger Population – Incorporating Molecularly Targeted Agents”

Authors: Keith Pratz, Harry Erba.

Frontline Therapy of AML in the Fit and Younger Population - Incorporating Molecularly Targeted Agents

Recent advances in acute myeloid leukemia (AML) treatment highlight the success of BCL-2 inhibition with venetoclax. The Viale-A phase 3 trial showed that venetoclax combined with azacitidine improved overall survival (OS) to 14.7 months versus 9.6 months with placebo, with 42% of patients achieving measurable residual disease (MRD)-negative complete remission (CR).

Other combinations, like venetoclax with gilteritinib, achieved a 96% complete remission (CR) rate and 93% MRD negativity, with 18-month OS and relapse-free survival (RFS) rates of 72% and 71%, respectively.

Venetoclax has also been incorporated into higher intensity regimens, such as FLAG-IDA, achieving a CR rate of 73% and a 24-month OS of 76%. Studies with venetoclax and standard chemotherapy regimens reported CR rates up to 91%, with high MRD negativity.

In post-transplant settings, FLT3 inhibitors, such as gilteritinib, improved relapse-free survival for FLT3-ITD AML patients with detectable MRD.

Measurable residual disease (MRD) is a critical factor in predicting AML outcomes. MRD-negative remission correlates with better prognosis, and future studies will define its role in guiding stem cell transplant decisions. The integration of targeted therapies with traditional chemotherapy is improving survival and remission rates, potentially reshaping AML treatment.

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