Results of the 6-Year Follow-up of the Gimema AML1310 Trial: A Risk-Adapted, MRD-Directed Therapy for Young Adults with Newly Diagnosed Acute Myeloid Leukemia

Background: In the AML1310 trial, we applied a comprehensive AML risk assessment, based on the integration of cytogenetic/genetic data and measurable residual disease (MRD) status, to optimize patients' (pts) therapeutic post-remission allocation. By doing so and using the NCCN2009 risk-stratification, favorable-risk (FR) pts (NPM1 mut/FLT3-ITD wt or CBF positive without c-Kit mutations) were to receive an autologous stem cell transplant (AuSCT); poor-risk (PR) pts (adverse karyotype or FLT3-ITD mut) were to receive an allogeneic stem cell transplant (ASCT); intermediate-risk (IR) pts (intermediate karyotype or FLT3-TKD mut or CBF positive with c-Kit mut) were to receive AuSCT or ASCT depending on the levels of MRD, measured by flow cytometry after consolidation therapy. Allocation to ASCT required the procedure to be performed whatever the source of stem cells (identical sibling, unrelated, cord blood, haploidentical). At that stage of analysis, 2-year overall (OS) and disease-free survival (DFS) of the whole series was 56% and 54%, respectively. Two-year OS and DFS were 74% and 61% in the FR category, 42% and 45% in the PR category, 79% and 61% in the IR MRD-negative category, 70% and 67% in the IR MRD-positive category (Venditti, Blood 2019:134(12);935-945) .