A new analysis by researchers at The University of Texas MD Anderson Cancer Center demonstrates that combination therapy consisting of fludarabine, cytarabine and G-CSF (FLAG) plus gemtuzumab ozogamicin (GO) or idarubicin (IDA) continues to deliver strong long-term outcomes for patients with core-binding factor acute myeloid leukemia (CBF-AML), a subtype of the disease involving a chromosomal rearrangement.
The study, published in Blood Cancer Discovery, was co-led by Gautam Borthakur, M.D., professor of Leukemia, and Jayastu Senapati, M.D., assistant professor of Leukemia. The researchers analyzed long-term outcomes from a cohort of patients treated in a Phase 2 trial.
The five-year overall survival rate reached 74%, while the relapse-free survival rate was 67% for patients treated in this study. Among patients who had received GO along with FLAG (FLAG-GO), the five-year overall survival rate was superior at 80% as opposed to FLAG-IDA on non-randomized comparisons.
“This is one of the highest reported five-year, relapse-free overall survival rates we have observed,” Borthakur said. “This regimen is now our standard frontline therapy for adults with core-binding factor AML, and these findings further strengthen the evidence supporting its use.”
Why are these findings significant?
CBF‑AML is considered a favorable‑risk leukemia, but relapse remains a major challenge. The ability to demonstrate high, five-year overall survival rates with FLAG-based therapy, particularly FLAG-GO, confirms this regimen is safe and provides durable efficacy, according to the researchers.
The Phase 2 clinical trial treated 219 newly diagnosed patients at UT MD Anderson. Researchers also examined whether baseline concurrent myeloid mutations influenced treatment outcomes. The study showed that these mutations including KIT mutations, which traditionally have been considered adverse — did not impact survival outcomes and the dynamics of deep molecular responses achieved by the patients.
