Children's Oncology Group's 2013 blueprint for research: Acute myeloid leukemia

Abstract

For the 365 children diagnosed with acute myeloid leukemia in the US annually, 5-year survival for patients on COG trials with low, intermediate, and high risk disease is 83%, 62%, and 23%, respectively. Recent advances include improved therapeutic stratification, improved survival with dose intensification, and further elucidation of the heterogeneity specific to childhood AML. These discoveries now guide current strategy incorporating targeted agents to pathways specific to childhood AML as well as evaluating methods to increase the sensitivity of the leukemic stem cell, first in Phase II feasibility trials followed by Phase III efficacy trials of the most promising agents. Acute myeloid leukemia in children, though with similar subgroups to adults, remains uniquely different based upon quite different prevalence of subtypes as well as overall response to therapy. The Children's Oncology Group's research agenda builds upon earlier efforts to better elucidate the leukemogenic steps distinct to childhood AML in order to more scientifically develop and test novel therapeutic approaches to the treatment and ultimate cure for children with this disorder.

Progress and Prospects in Pediatric Leukemia

“…62 But outcomes for high-risk patients still remain poor with an EFS ranging from 15 to 35% and a maximum OS of 46%. Therapy-related AML has relatively poor outcomes as compared to de novo AML, with survival rates around 20%.…”

Administration of Dexrazoxane Improves Cardiac Indices in Children and Young Adults With Acute Myeloid Leukemia (AML) While Maintaining Survival Outcomes

“…1–4 Pediatric cancer survivors who received intensive treatment, such as those with AML, have a 73% incidence of developing a chronic health condition and 42% chance of having a severe, disabling, life-threatening condition or death from a chronic condition. 5 Importantly the absence of a plateau in chronic condition incidence over 30 years of follow-up is most concerning.…”

Antibiotic prophylaxis with teicoplanin on alternate days reduces rate of viridans sepsis and febrile neutropenia in pediatric patients with acute myeloid leukemia

“…Cure rates are lower in comparison to those in acute lymphoblastic leukemia [1]. Yet, intensive chemotherapy based mainly on anthracyclines and purine analogues has improved outcome over the last decades [1–3]. Profound and prolonged neutropenia following intensive chemotherapy cycles, however, is associated with a high risk for bacterial and fungal infections contributing to considerable therapy-associated morbidity and mortality [4, 5].…”