Pediatric acute myeloid leukemia: updates on biology, risk stratification, and therapy

Despite advances in therapy over the past decades, overall survival for children with acute myeloid leukemia (AML) has not exceeded 70%. In this review, we highlight recent insights into risk stratification for patients with pediatric AML and discuss data driving current and developing therapeutic approaches.

Advances in cytogenetics and molecular profiling, as well as improvements in detection of minimal residual disease after induction therapy, have informed risk stratification, which now relies heavily on these elements. The treatment of childhood AML continues to be based primarily on intensive, conventional chemotherapy. However, recent trials focus on limiting treatment-related toxicity through the identification of low-risk subsets who can safely receive fewer cycles of chemotherapy, allocation of hematopoietic stem-cell transplant to only high-risk patients and optimization of infectious and cardioprotective supportive care.

Further incorporation of genomic and molecular data in pediatric AML will allow for additional refinements in risk stratification to enable the tailoring of treatment intensity. These data will also dictate the incorporation of molecularly targeted therapeutics into frontline treatment in the hope of improving survival while decreasing treatment-related toxicity.