No abstract

Intensive, myelosuppressive therapy is necessary to maximize outcomes for patients with acute myeloid leukemia (AML). A comparison was made of 3 aggressive postremission approaches for children and adolescents with AML in a randomized trial, CCG-2891. A total of 652 children and adolescents with AML who achieved remission on 2 induction regimens using identical drugs and doses (standard and intensive timing) were eligible for allocation to allogeneic bone marrow transplantation (BMT) based on matched related donor status (n ‫؍‬ 181) or randomization to autologous BMT (n ‫؍‬ 177) or to aggressive high-dose cytarabine-based chemotherapy (n ‫؍‬ 179).Only 115 patients (18%) refused to participate in the postremission phase of this study. Overall compliance with the 3 allocated regimens was 90%. At 8 years actuarial, 54% ؎ 4% (95% confidence interval) of all remission patients remain alive. Survival by assigned regimen ("intent to treat") is as follows: allogeneic BMT, 60% ؎ 9%; autologous BMT, 48% ؎ 8%; and chemotherapy, 53% ؎ 8%. Survival in the allogeneic BMT group is significantly superior to autologous BMT (P ‫؍‬ .002) and chemotherapy (P ‫؍‬ .05); differences between chemotherapy and autologous BMT are not significant (P ‫؍‬ .

This trial demonstrated that early response assessment supported therapeutic titration (omitting radiotherapy in RERs with CR; augmenting chemotherapy in SERs with PET-positive disease). Strategies directed toward improved response assessment and risk stratification may enhance tailoring of treatment to patient characteristics and response.