No Easy Target! What’s new in AML Therapy? (225)

The suboptimal outcomes that exist for AML are likely because of the gene mutations and rearrangements present in this disease (Conneely & Stevens, 2021; Lonetti et al, 2019). The acute and chronic toxicities of current chemotherapy regimens have opened the door to alternative treatment approaches. In the last several years, our understanding of AML biology and the molecular pathways has increased and has generated the development of novel drugs to target these specific pathways (Chen & Glasser, 2020). Intensified treatment approaches, improvements in supportive care, and progress in disease stratification have resulted in improvements in overall survival (OS) in the last 20 years . Despite those enhancements, OS does not exceed 70% and relapse rates remain at 25%–35%. This intensifies the need for new therapeutic strategies, such as novel targeted treatments (Conneely & Stevens, 2021; Lonetti et al., 2019). This presentation will work to bridge the gap between the advancements in targeted therapies for pediatric AML patients and the practice knowledge of the pediatric oncology nurse, the advanced practice nurse, and the pharmacist. We will provide a high-level overview of targeted therapies, including drugs that recently received approval by the U.S. Food and Drug Administration and novel therapies currently being studied in clinical trials, to set the stage for the treatment advancements in pediatric AML. We also will share a review of The Leukemia and Lymphoma Society’s Children’s Initiative, including the global precision medicine master clinical trial PedAL.