De Novo AML

Overall survival in childhood and young adult acute myeloid leukemia (AML) has improved significantly over the last 40 years, largely due to intensification of chemotherapy, improved identification of patients with higher risk of relapse, advances in hematopoietic stem cell transplantation, and improved supportive care (Zwaan et al., J Clin Oncol 33(27):2949–2962, 2015; Rasche et al., Leukemia 32(10):2167–2177, 2018). The pillars of chemotherapy are largely unchanged, and center around cytarabine and anthracyclines. As we improve our biologic understanding of childhood AML, we have been able to better determine which patients are at the highest risk of recurrence and in some cases identify targets for which there are emerging therapeutics. However, due to the combination of complex biologic heterogeneity and the rare incidence of childhood and young adult AML, the importance of large-scale international cooperative groups to conduct clinical and biologic research becomes more critical. This chapter will focus on therapeutic advances in childhood AML in the international cooperative groups, including chemotherapeutic regimens and advances in risk stratification.