A new analysis by researchers 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 leukaemia (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 Leukaemia, and Jayastu Senapati, M.D., assistant professor of Leukaemia.

The researchers analysed 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-randomised 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 favourable‑risk leukaemia, 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.

Article: Integrated analysis of genomics, molecular responses and outcomes of CBF-AML with FLAG based therapy on a phase 2 trial

Source: University of Texas M. D. Anderson Cancer Center