Childhood Cancer

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Characterizing the role of measurable residual disease (MRD) in pediatric acute myeloid leukemia (AML) survival disparities

Institution: 
Children’s Hospital of Philadelphia
Researcher(s): 
Daniel Zheng, MD
Grant Type: 
Young Investigator Grants
Year Awarded: 
2025
Type of Childhood Cancer: 
Acute Myeloid Leukemia (AML)
Project Description: 

Acute myeloid leukemia (AML) is a rare but very serious type of blood cancer in children. While treatments for childhood cancer have improved, 20-40% of children of AML still do not survive. Black and Hispanic children are less likely to survive than White children, which may be due to differences in access to care, delays in getting diagnosed, or other barriers that disproportionately affect families from minority communities. Black and Hispanic children are also less likely to be enrolled on clinical trials, but children on clinical trials do better. We don’t know why. Doctors use a blood test called measurable residual disease (MRD) to see how well a child is responding to their first round of chemotherapy. MRD looks for tiny amounts of cancer cells left in the body after treatment, and children who don’t have any detectable cancer cells are more likely to survive. We don’t know if MRD is different across racial and ethnic groups or if it explains why children in clinical trials tend to have better survival rates than those who aren’t in trials. One of the things we think may influence MRD is how sick a child is when they are first diagnosed, which may reflect decreased access health care and a delay in diagnosis. Our prior research showed that Black and Hispanic children are sicker compared to White children when they are first diagnosed and that sicker children seem less likely to enroll on clinical trials.

Project Goal:

This project overall aims to better understand why some children with AML do worse than others so that we can improve outcomes for everyone. We will look at whether MRD plays a role in survival differences between racial and ethnic groups and between children who are and aren’t enrolled in clinical trials. We’ll also explore whether being very sick at diagnosis contributes to these differences. To do this, we’re working with 15 different children’s hospitals across the country to study a large group of children with AML (around 1,300). We will collect detailed information from their medical record about their AML, how sick they were at diagnosis, their MRD response after chemotherapy, and whether they survived. This research hopes to uncover ways to provide earlier and better care, reduce survival gaps, and ensure that every child with AML has a better chance to beat cancer.