Mentor Name: Richard Aplenc

Pediatric AML is the second most common pediatric hematologic malignancy and requires very intensive, typically inpatient therapy. Recent efforts to improve outcomes in pediatric AML have focused in large part in testing novel therapies in genomically defined patient subgroups. FLT3 mutated AML is a well described genetic subset that affects approximately 20% of pediatric AML patients. The Children’s Oncology Group (COG) recently published data strongly suggesting that sorafenib improves outcomes for children with high allelic ratio FLT3 ITD (PMID: 35349331). However, these analyses have important limitations, specifically the requirement for clinical trial enrollment and the exclusion of individuals with low allelic ratio FLT3 ITD and tyrosine kinase domain (TKD) mutations. Given this data gap, we seek to determine the real-world efficacy of sorafenib as well its efficacy in patients with low allelic ratio FLT3 ITD and TKD mutations. Dr. Aplenc, in collaboration with Dr. Kelly Getz, has developed a real-world AML chart abstraction cohort that includes over 1,000 patients. This cohort includes patients diagnosed with AML at 17 institutions across the United States between 2011-2024 and currently includes approximately 1,000 patients. During this summer research experience, we will perform manual chart abstraction to expand this cohort to more than 1,400 patients. This data collection will include clinical outcome data and genomic data from diagnosis and relapse. These data will then be used to support the comparative effectiveness analyses using an emulated clinical trial framework.