Clinical Epidemiology Evaluations of Treatment Outcomes in Children with Acute Myeloid Leukemia
Background
Over the past several decades, overall survival rates for pediatric acute myeloid leukemia (AML) improved to nearly 65% through intensive chemotherapy regimens and enhanced supportive care. Anthracyclines contributed significantly to these improvements, but are also associated with substantial cardiotoxicity. Children with AML receive the highest cumulative anthracycline doses among all frontline pediatric cancer therapies, putting them at a particularly high risk for cardiovascular injury. Each treatment course also results in a period of prolonged neutropenia during which patients are highly susceptible to severe infection, a major cause of therapy-related morbidity and mortality. Although pediatric cancer supportive care guidelines recommend that patients remain hospitalized until resolution of neutropenia, some hospitals discharge their patients to be managed at home during neutropenia after chemotherapy, while others strictly utilize inpatient management. Published data evaluating the benefits of cardio-protective agents during AML treatment generally have not included AML patients. Additionally, there is limited literature on the clinical consequences of outpatient versus inpatient management, making it difficult to determine the best way to manage patients during periods of neutropenia.
Project Goal
Chelsea Young will provide research support to two studies addressing these knowledge gaps: the first is an ALSF-funded study aimed at assessing the potential utility of dexrazoxane as a cardio-protective intervention in pediatric AML; the second is a Patient Center Outcomes Research Institute (PCORI) study aimed at comparing clinical outcomes, including the occurrence of infection and treatment delays, as well as the patient’s quality of life for at-home relative to inpatient management of neutropenia after chemotherapy for pediatric AML.
Mentored by Dr. Kelly Getz
Children’s Hospital of Philadelphia, Philadelphia, PA
