AAML1531, Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

in summary, the proposed study aaML1531 builds on the success of the initial steps taken by two previous CoG studies, a2971 and aaML0431; implements MRD-based risk stratification of DS aML by eliminating HD ara-C advances aML therapy for children with DS in the direction of treatment reduction for the majority of patients. in addition, for the first time, this protocol provides an intensified therapeutic strategy targeted to high risk patients with DS aML, for whom there are currently neither specific treatment studies nor treatment recommendations. Thus, aaML1531 achieves risk-adapted stratification of treatment for DS aML as a rational and necessary next step of trial design for this vulnerable population.