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Race and Ethnicity and Overall Survival in Pediatric Acute Myeloid Leukemia
Daniel J Zheng1,2, Long Khuong3, Catherine Aftandilian4
1Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia.
Insights
Black and Hispanic children with acute myeloid leukemia (AML) have worse survival rates than White children. Cardiovascular and respiratory issues at diagnosis partially explain these disparities in pediatric AML survival.
Area of Science:
- Pediatric Oncology
- Health Disparities Research
- Epidemiology
Background:
- Historically, Black and Hispanic children diagnosed with acute myeloid leukemia (AML) have faced poorer survival outcomes compared to their White counterparts.
- Identifying the specific intermediate factors contributing to these disparities is crucial for targeted interventions.
Purpose of the Study:
- To compare survival outcomes in a large contemporary cohort of pediatric AML patients across racial and ethnic groups.
- To investigate presentation acuity, frontline organ toxicities, and relapse rates as potential mediators of observed survival differences.
Main Methods:
- A retrospective cohort study analyzed data from 773 children with de novo AML treated at 13 US children's hospitals from January 2011 to May 2024.
- Overall survival (OS) was the primary outcome, with analyses examining presentation acuity, organ toxicities, and relapse rates stratified by race and ethnicity.
Main Results:
- Black and Hispanic children exhibited worse 5-year overall survival (61.1% and 65.4%, respectively) compared to White children (70.0%).
- Black patients showed increased risk of higher cardiovascular, respiratory, and renal acuity at presentation.
- Cardiovascular and respiratory acuity at presentation were independently associated with all-cause mortality and partially mediated survival differences between Black and White children.
Conclusions:
- Differential cardiovascular and respiratory acuity at presentation partially explain survival outcome disparities for Black children with AML.
- Further research is needed to identify other intermediate factors along the care continuum to fully account for survival differences and guide interventions.
Importance:
Hispanic and non-Hispanic Black (hereafter, Black) children with acute myeloid leukemia (AML) have historically experienced worse survival outcomes compared with non-Hispanic White (hereafter, White) children. Understanding specific intermediate pathways underlying these outcome disparities is a critical step to directing interventional efforts and resources.
Objective:
To compare survival outcomes by race and ethnicity in a large contemporary cohort of pediatric patients with AML and to examine presentation acuity, frontline organ toxic effects, and relapse as potential mediators of any observed outcome disparities.
Design, Setting, And Participants:
This retrospective cohort study was analyzed from April 2025 to January 2026 and included 13 US children's hospitals across 10 states participating in the Real-World Epidemiology of Acute Leukemia-AML cohort. Participants included children treated for de novo AML from January 2011 through May 2024.
Exposure:
Race and ethnicity (Black, Hispanic, and White).
Main Outcomes And Measures:
The main outcome was overall survival (OS), defined as the time from AML diagnosis until death from any cause.
Results:
The study cohort included 773 children (median [IQR] age, 9 [2-14] years; 402 males [52.0%]), of whom 125 (16.2%) were Black, 237 (30.7%) were Hispanic, and 411 (53.2%) were White (median [IQR] follow-up, 51.2 [25.7-86.9] months), treated for de novo AML. Among the patients, Black (5-year OS, 61.1% [95% CI, 52.6%-71.0%]; adjusted hazard ratio [AHR], 1.55 [95% CI, 1.26-1.91]) and Hispanic (5-year OS, 65.4% [95% CI, 58.9%-72.5%]; AHR, 1.32 [95% CI, 1.03-1.68]) children had worse OS compared with White children (5-year OS, 70.0% [95% CI, 65.2%-75.1%]). Compared with White patients, Black patients had increased risk of higher cardiovascular (adjusted risk ratio [ARR], 2.09 [95% CI, 1.23-3.56]), respiratory (ARR, 1.36 [95% CI, 1.05-1.77]), and renal (ARR, 3.08 [95% CI, 1.02-9.32]) acuity at presentation. There were no statistically significant increases in severe frontline organ toxic effects or relapse rates by race and ethnicity. Cardiovascular acuity (AHR, 1.83 [95% CI, 1.51-2.22]) and respiratory acuity (AHR, 1.43 [95% CI, 1.15-1.77]) at presentation were independently associated with all-cause mortality. Cardiovascular acuity (proportion mediated, 14%) and respiratory acuity (proportion mediated, 8%) at presentation partially mediated the total absolute survival difference between Black and White children.
Conclusions And Relevance:
In this cohort study of pediatric patients with AML, differential cardiovascular and respiratory acuity at presentation were partial mediators of outcome disparities for Black children with AML. Other intermediates along the care continuum should be identified to account for the remainder of the observed outcomes and to prioritize focus areas for interventional efforts to mitigate the survival differences.
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