AAML1531 - Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome. A COG Groupwide Phase 3 Study (CTMS# 17-0058)
1.1 Primary Aims
1.1.1 To determine the 2-year event-free-survival (EFS) for children with standard risk DS AML (MRD-negative after one cycle of induction therapy) after elimination of HD Ara-C from the treatment regimen.
1.1.2 To determine the 2-year EFS for children with high risk DS AML (MRD-positive after one cycle of induction therapy) after intensification of treatment equivalent to that used for high risk AML in children without DS.
1.2 Exploratory Aims
1.2.1 To determine the extent to which elimination of HD Ara-C from the treatment of standard risk DS AML decreases adverse events and resource utilization. Specifically, to determine if elimination of HD Ara-C from treatment of standard risk DS AML results in:
220.127.116.11 A significant decrease in the number of days per patient spent on protocol therapy compared to predecessor study AAML0431.
18.104.22.168 A significant decrease in the average number of days of hospitalization per patient compared to predecessor studies AAML0431 and A2971.
22.214.171.124 A significant decrease in the number (per patient) and rate (per duration of treatment) of sterile site infections compared to the predecessor study AAML0431.
126.96.36.199 A significant decrease of resource utilization by AML treatment compared to the predecessor study AAML0431.
1.2.2 To compare the feasibility and analytical characteristics of flow cytometry, PCR and targeted error-corrected sequencing of GATA1 mutations as methods to detect MRD in DS AML.
1.2.3 To establish a DS AML cell bank of viably frozen bone marrow samples collected at the end of induction and corresponding non-tumor DNA samples collected at end of Induction 1.