Mentor Name: Michael Pulsipher

The project goal is to collect data in a standardized manner from multiple pediatric cancer centers to develop clinical guidance for improving the care of children, adolescents, and young adults (AYA) with newly diagnosed and relapsed acute myeloid leukemia (AML). Currently, there is no chemotherapy regimen considered standard of care for treating pediatric AML patients who are newly diagnosed or relapsed. Physicians lack real-world data for supporting clinical treatment decisions, particularly around supportive care measures to manage short-term and long-term toxicities, such as infectious burdens and cardiotoxicities, which can significantly impact the life of a child and family. Selecting a chemotherapy regimen may rely on institutional experience or expert opinion, both of which may be biased. For this project, we intend to utilize and expand on the multi-institutional cohort of pediatric AML patients at 19 institutions across the United States initially created through the Patient-Centered Outcomes Research Institute award to Dr. Richard Aplenc and Dr. Kelly Getz. The cohort was initially developed to investigate if AML patients can be safely discharged home after completion of chemotherapy, but there are ongoing efforts to expand the cohort to continue to answer clinically meaningful questions in pediatric AML care. To create a robust database, the data abstraction is extensive and standardized. The comprehensive data collection includes demographic, leukemia characteristics, chemotherapy treatment, toxicity, heart function, clinical trial participation, treatment response, minimal residual disease, relapse, and death data. Extensive in-person training and weekly virtual support is conducted by the Children’s Hospital of Philadelphia study team. Data collection is extensive, and it takes approximately 3-4 hours per patient to complete abstraction. The real-world cohort is intended to be geographically representative. As the only free-standing children’s hospital in the Intermountain West with a catchment area that covers one-seventh of the United States by land mass and encompassing ~400,000 square miles, we feel the contribution of the data from Primary Children’s Hospital/University of Utah is essential to ongoing efforts in care for pediatric and AYA patients diagnosed with AML. Overall, the continued development and maintenance of a real-world dataset for childhood AML will allow for a comparison of outcomes including treatment response and side effects between chemotherapy regimens for AML. This information will be valuable to clinicians who care for AML patients and impact the care of children, adolescents, and young adults with AML.