A Danish nationwide population-based cohort study on acute myeloid leukemia with RUNX1::RUNX1T1 – Real-world outcomes and clinicopathological characteristics

Le résumé fourni par la source

Core-binding factor (CBF) acute myeloid leukemia (AML) with the RUNX1::RUNX1T1 fusion oncogene resulting from the recurrent balanced translocation t(8;21)(q22;q22.1) was among the first acute leukemias to be defined based on cytogenetics [ 1 ]. It has been estimated to account for 7% of all AML cases [ 2 , 3 ] and is associated with a favorable prognosis [ 4 ]. The addition of gemtuzumab ozagamicin (GO) to intensive chemotherapy was initially reported to improve both relapse-free survival (RFS) and overall survival (OS) in patients with favorable cytogenetic profiles [ 5 , 6 , 7 ]. However, multiple randomized clinical trials failed to demonstrate an OS benefit upon final analysis [ 8 , 9 , 10 , 11 , 12 ]. Additionally, relapse rates of up to 40% have been reported, significantly reducing survival [ 13 , 14 ]. Data regarding the precise risk of relapse and long-term outcomes for patients with RUNX1::RUNX1T1 CBF-AML remains warranted at a population level. We conducted a population-based characterization of clinical- and molecular features and analyzed long-term outcomes for RUNX1::RUNX1T1 CBF-AML patients.

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