CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS Results of consecutive trials for children newly diagnosed with acute myeloid leukemia from the Australian and New Zealand Children’s Cancer Study Group

Despite improvements in the treatment of acute myeloid leukemia (AML), approximately 50% of children die of the disease. Clinical trials in adult patients with AML indicate that idarubicin may have superior efficacy when compared to daunorubicin in the remission-induction phases of chemotherapy. We conducted consecutive clinical trials in children with newly diagnosed AML in which daunorubicin (group 1, n 102) or idarubicin (group 2, n 160) was used during the remissioninduction (RI) and the early consolidation phases of chemotherapy. Idarubicin was given at a dose of either 10 mg/m2 (group 2A, n 106) or 12 mg/m2 (group 2B, n 53). A high rate of RI was achieved for all groups (95% group 1, 90% group 2A, 94% group 2B). There were no significant differences in 5-year event-free survival (EFS) or in overall survival (OS) when the 3 groups were compared (group 1: EFS 50%, OS 56%; group 2A: EFS 50%, OS 60%; group 2B: EFS 34%, OS 50%). RI deaths resulting from treatment toxicity were low—2% for group 1 and 5% for group 2. More gastrointestinal, pulmonary, and renal toxicity but fewer infections were observed in patients receiving idarubicin (P < .001, P .04, P .03, respectively). Following RI chemotherapy, all patients received 3 to 4 more courses of identical chemotherapy and then underwent either autologous (n 156) or an allogeneic bone marrow transplantation (BMT) (n 35). OS was higher in allogeneic BMT patients than in autologous BMT patients (79% vs 63%; P .23). We conclude that daunorubicin is as effective as idarubicin for remission-induction therapy for childhood AML and has reduced toxicity. (Blood. 2002;100: 2708-2716)