Treatment of Acute Myeloid Leukemia in Core Binding Factor AML (RUNX1-RUNX1T1 or CBFB-MYH11-Positive)

Core binding factor AML (CBF-AML) is defined by the presence of either the RUNX1-RUNX1T1 or CBFB-MYH11 fusion. This genetically distinct subtype has an established first-line treatment pathway for patients eligible for standard-intensity induction and consolidation chemotherapy.

Clinical scenario

Patients with confirmed CBF-AML — RUNX1-RUNX1T1-positive or CBFB-MYH11-positive — who are fit for standard induction and consolidation chemotherapy. CD33 expression on leukaemic blasts is relevant to treatment selection within this population.

Treatment approach — first-line induction (partial overview)

Induction incorporates a cytarabine-based backbone combined with an anthracycline. When leukaemic blasts are CD33-positive, an additional targeted agent is incorporated into the induction regimen alongside the standard doublet.

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Treatment goals

The primary goal is achievement of complete remission (CR) or CR with incomplete count recovery (CRi), defined as fewer than 5% blasts in the bone marrow. Response is assessed between day 14 and day 21 following the first induction cycle.

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References

For CBF-AML, the authors recommend 7 days of cytarabine, 3 days of daunorubicin (7+3) and 1–3 days of gemtuzumab ozogamicin (GO) in induction 1: 7+3+GO [II, A].
GO is approved as a fractionated dose of 3 mg/m² on days 1, 4 and 7 based on the ALFA-0701 trial.
After the first induction cycle, response should be assessed between day 14 and day 21.
As soon as patients achieve CR/CRi after 1 or 2 induction cycles, they should proceed to consolidation treatment [II, B].

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