Less-intensive drug combination is more effective in treating acute myeloid leukemia, clinical trial finds

Investigators at the Mass General Brigham Cancer Institute have discovered that a less-intensive drug combination of azacitidine and venetoclax proves more effective in treating acute myeloid leukemia (AML) compared to intensive chemotherapy. Published in the New England Journal of Medicine, these findings may alter the initial treatment approach for many AML patients.

Lead author Dr. Amir T. Fathi emphasized that the goal is to optimally treat AML while minimizing serious complications and hospital stays. The less-intensive combination, already utilized for older or less tolerant patients, was tested in the PARADIGM trial, comparing its efficacy to intensive chemotherapy in 172 eligible adults.

Results showed that patients receiving azacitidine and venetoclax experienced a 14.5-month progression-free survival, compared to 6.2 months for those receiving intensive chemotherapy. Additionally, the combination resulted in remission for 78% of patients, versus 53% for intensive chemotherapy, fewer serious infections and bleeding incidents, and a need for fewer hospitalizations, with patients spending 12.5 days in the hospital with the less-intensive regimen versus 27.3 days with intensive chemotherapy.

Notably, 60% of patients on the gentler treatment proceeded to stem cell transplantation, compared to 40% on intensive chemotherapy. While the trial did not evaluate overall survival, future studies should explore the less-intensive approach in younger AML patients with specific genetic mutations.