Data driven response criteria in pediatric acute myeloid leukemia
Mentor Name: Richard Aplenc
Pediatric acute myeloid leukemia (AML) is the second most common pediatric hematologic malignancy and requires very intensive, typically in-patient therapy. The development of new therapies to improve AML outcomes is challenged by current guidance from the Food and Drug Administration (FDA) that requires patients to achieve neutrophil and platelet recovery thresholds based exclusively on historical adult data. These thresholds of a neutrophil count greater than 1000/ul and a platelet count of greater than 100,000/ul are not used in Children’s Oncology Group (COG) clinical trials and are rarely met in current pediatric clinical practice. The FDA requires attainment of these adult thresholds to qualify for complete response, the criteria used to determine clinical effectiveness for FDA approval of novel therapies. Thus, the lack of data-driven pediatric standards hinders the ability of investigators and industry sponsors to develop new therapies for pediatric AML. This project will contribute to manual chart abstraction for a multi-institution cohort of pediatric AML patients that will develop the data necessary for establishing a data-driven pediatric AML response standard. This data collection will include clinical outcome data and a 14-day window of neutrophil and platelet count data at the end of induction therapy in the newly diagnosed and relapse AML settings. These data will then be used to evaluate the current adult thresholds as well as the current COG threshold used in front-line COG clinical trials.
