Real-world generalizability of clinical trial cytomolecular risk in pediatric acute myeloid leukemia: a report from the REAL-AML cohort
Cytomolecular features critical for risk-stratified treatment determination in pediatric acute myeloid leukemia (AML) were expanded in Children's Oncology Group (COG) Phase III trial AAML1831 based on previous trials. It remains unknown whether the cytomolecular risk profiles are generalizable to the real-world. We addressed this knowledge gap using a nationally representative real-world cohort of 913 pediatric AML patients. Distributions of cytomolecular risk profiles and individual markers were comparable for trial-enrolled and non-enrolled patients, as well as across social drivers of trial enrollment (race/ethnicity, language, insurance, acuity). Compared to patients with only favorable cytomolecular markers (4-year OS 89.48%; 95% CI: 84.46%-92.95%), patients with both favorable and unfavorable (hazards ratio [HR] = 2.49, 95% CI : 1.18-5.23), neutral (HR = 4.33, 95% CI : 2.75-6.82), and only unfavorable (HR = 5.80, 95% CI: 3.70-9.11) markers all had increased hazards of death. Cytomolecular risk informed by trial data appears to be generalizable to the real-world setting in pediatric AML.
Authors
- Elaine R. Morgan (ORCID: https://orcid.org/0000-0003-2505-8885)
- Rajen J. Mody (ORCID: https://orcid.org/0000-0001-5574-1779)
- Craig Lotterman
- Yimei Li (ORCID: https://orcid.org/0000-0001-9067-6992)
- Daniel J. Zheng (ORCID: https://orcid.org/0000-0003-2217-088X)
- Emi Caywood (ORCID: https://orcid.org/0000-0002-2873-6058)
- Caroline Smith (ORCID: https://orcid.org/0000-0002-9154-6855)
- Tamara P. Miller (ORCID: https://orcid.org/0000-0003-4250-5376)
- Richard Aplenc (ORCID: https://orcid.org/0000-0001-7482-5644)
- Haley Newman (ORCID: https://orcid.org/0000-0002-3022-8052)
- Alix E. Seif (ORCID: https://orcid.org/0000-0002-1799-2582)
- Jennifer Jill Wilkes (ORCID: https://orcid.org/0000-0002-3806-6019)
- Jamie Stokke
- Maria Monica Gramatges (ORCID: https://orcid.org/0000-0002-0947-104X)
- Cody Gathers
- Naomi Joan Winick (ORCID: https://orcid.org/0000-0002-6636-3870)
- Anderson B. Collier (ORCID: https://orcid.org/0000-0001-7183-7826)
- Caitlin W. Elgarten (ORCID: https://orcid.org/0000-0002-2474-3031)
- Xin Wang (ORCID: https://orcid.org/0000-0001-9827-3098)
- Kelly Getz (ORCID: https://orcid.org/0000-0003-2020-5153)
- Kelly W. Maloney (ORCID: https://orcid.org/0000-0002-3711-9529)
- Meret Henry (ORCID: https://orcid.org/0000-0001-5612-3294)
- Gary Hettinger (ORCID: https://orcid.org/0000-0001-5980-2946)
- Yuan‐Shung Huang (ORCID: https://orcid.org/0000-0002-1942-6197)
- Kira O'Neil Bona (ORCID: https://orcid.org/0000-0001-5602-7059)
- Arunkumar Modi (ORCID: https://orcid.org/0000-0002-9323-8979)
- Regina M. Myers (ORCID: https://orcid.org/0000-0002-2542-2061)
- Catherine C. Aftandilian (ORCID: https://orcid.org/0000-0001-9775-2839)
- Taumoha Ghosh (ORCID: https://orcid.org/0000-0003-3420-3167)
- José Ortiz
- Victor Wong (ORCID: https://orcid.org/0000-0002-5254-0039)
- Amir Mian (ORCID: https://orcid.org/0009-0005-1178-727X)
Institutions
- Arkansas Children's Hospital (US)
- Primary Children's Hospital (US)
- Memorial Sloan Kettering Cancer Center (US)
- Children's Hospital of Philadelphia (US)
- Children's Hospital of Los Angeles (US)
- Thomas Jefferson University (US)
- Emory University (US)
- Palo Alto University (US)
- Baylor College of Medicine (US)
- University of Michigan (US)
- Ochsner Medical Center (US)
- Rady Children's Hospital-San Diego (US)
- Children's Hospital Colorado (US)
- Dell Children's Medical Center of Central Texas (US)
- Nemours Children's Health System (US)
- Seattle Children's Hospital (US)
- Aflac (United States) (US)
- Michigan Medicine (US)
- Dana-Farber Cancer Institute (US)
- Lurie Children's Hospital (US)
- Children's Hospital of Michigan (US)
- Stanford Medicine (US)
- Children's Healthcare of Atlanta (US)
- Texas Children's Hospital (US)
- New York University (US)
- University of Pennsylvania (US)
- The University of Texas Southwestern Medical Center (US)
- University of Colorado Denver (US)
- Stanford University (US)
Publication Details
- Journal
- JNCI Journal of the National Cancer Institute
- Published
- 2025-08-20
- DOI
- https://doi.org/10.1093/jnci/djaf234
- Primary Topic
- Childhood Cancer Survivors' Quality of Life
- Type
- article
- Field-Weighted Citation Impact
- 0.00