Real-world generalizability of clinical trial cytomolecular risk in pediatric acute myeloid leukemia: a report from the REAL-AML cohort

Journal of the National Cancer Institute Pub Date : 2025-08-20 DOI:10.1093/jnci/djaf234

Daniel J Zheng, Gary Hettinger, Catherine Aftandilian, Kira Bona, Emi H Caywood, Anderson B Collier, Caitlin W Elgarten, Cody Gathers, Taumoha Ghosh, M Monica Gramatges, Meret Henry, Yuan-Shung V Huang, Yimei Li, Craig Lotterman, Kelly Maloney, Amir Mian, Tamara P Miller, Arunkumar Modi, Rajen Mody, Elaine Morgan, Regina Myers, Haley Newman, Jose Ortiz, Alix E Seif, Caroline Smith, Jamie Stokke, Xin Wang, Naomi Winick, Jennifer J Wilkes, Victor Wong, Richard Aplenc, Kelly D Getz

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Abstract

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.

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儿童急性髓性白血病临床试验细胞分子风险的真实世界普遍性：REAL-AML队列报告

在儿童肿瘤组（COG） III期试验AAML1831的基础上，扩大了对儿童急性髓性白血病（AML）风险分层治疗确定至关重要的细胞分子特征。目前尚不清楚细胞分子风险概况是否可推广到现实世界。我们通过913例儿科AML患者的全国代表性真实队列来解决这一知识差距。在试验入组和未入组患者中，以及在试验入组的社会驱动因素（种族/民族、语言、保险、敏锐度）中，细胞分子风险概况和个体标志物的分布具有可比性。与仅有有利细胞分子标志物的患者（4年OS 89.48%, 95% CI: 84.46%-92.95%）相比，有利和不利标志物的患者（风险比[HR] = 2.49, 95% CI: 1.18-5.23）、中性标志物的患者（HR = 4.33, 95% CI: 2.75-6.82）和仅不利标志物的患者（HR = 5.80, 95% CI: 3.70-9.11）的死亡风险均增加。通过试验数据得知的细胞分子风险似乎可以推广到儿童AML的现实环境中。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Journal of the National Cancer Institute

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