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.
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This study was funded by UT MD Anderson institutional funds. A full list of authors and their disclosures can be found with the paper in Blood Cancer Discovery .
Blood Cancer Discovery